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Strategy uses particles to target gene in specific immune cells

Using dual-energy X-ray absorptiometry to identify fat (white) in the body, researchers at Washington University School of Medicine in St. Louis found excessive fat in a mouse that had consumed a high-fat diet for six weeks (left). The mouse on the right ate the same diet, but the researchers blocked the activity of a gene in specific immune cells, resulting in that mouse not becoming obese.

Disabling a gene in specific mouse cells, researchers at Washington University School of Medicine in St. Louis have prevented mice from becoming obese, even after the animals had been fed a high-fat diet.

The researchers blocked the activity of a gene in immune cells. Because these immune cells called macrophages are key inflammatory cells and because obesity is associated with chronic low-grade inflammation, the researchers believe that reducing inflammation may help regulate weight gain and obesity.

The study is published May 1 in The Journal of Clinical Investigation.

Weve developed a proof of concept here that you can regulate weight gain by modulating the activity of these inflammatory cells, said principal investigator Steven L. Teitelbaum, MD, the Wilma and Roswell Messing Professor of Pathology & Immunology. It might work in a number of ways, but we believe it may be possible to control obesity and the complications of obesity by better regulating inflammation.

When people are obese, they burn fewer calories than those who are not obese. The same is true for mice. But according to co-first author Wei Zou, MD, PhD, an assistant professor of pathology & immunology, the researchers found that obese mice maintained the same level of calorie burning as mice that were not obese after the research team deleted the ASXL2 gene in the macrophages of the obese mice and, in a second set of experiments, after they injected the animals with nanoparticles that interfere with the genes activity.

Despite high-fat diets, the treated animals burned 45% more calories than their obese littermates with a functioning gene in macrophages.

Exactly why this prevented obesity in the mice isnt clear. Co-first author Nidhi Rohatgi, PhD, an instructor in pathology, said it appears to involve getting white fat cells which store the fat that makes us obese to behave more like brown fat cells which help to burn stored fat. The strategy is a long way from becoming a therapy, but it has the potential to help obese people burn fat at rates similar to rates seen in lean people.

A large percentage of Americans now have fatty livers, and one reason is that their fat depots cannot take up the fat they eat, so it has to go someplace else, Teitelbaum said. These mice consumed high-fat diets, but they didnt get fatty livers. They dont get type 2 diabetes. It seems that limiting the inflammatory effects of their macrophages allows them to burn more fat, which keeps them leaner and healthier.

Zou W, et al. Myeloid-specific Axsl2 deletion limits diet-induced obesity by regulating energy expenditure. The Journal of Clinical Investigation, May 1, 2020.

This work was supported by the National Heart, Lung and Blood Institute; the National Institute of Arthritis and Musculoskeletal and Skin Diseases; the National Institute of Diabetes and Digestive and Kidney Diseases; the National Institute of Allergy and Infectious Diseases and the National Institute of Biomedical Imaging and Bioengineering of the National Institutes of Health (NIH). Grant numbers HL1388163, AR064755, AR068972, AR070975, HL38180, DK56260, P30 DK52574, P41 EB025815, HL073646, DK102691, AI019653, DK109668, DK056341, AR046523, DK111389 and P30 AR074992. Additional funding from the Physician-Scientist Training Program at Washington University School of Medicine and the Childrens Discovery Institute.

Washington University School of Medicines 1,500 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

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Obesity prevented in mice treated with gene-disabling nanoparticles - Washington University School of Medicine in St. Louis

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Foundation Medicine, Inc. today announced that it has received approval from the U.S. Food and Drug Administration (FDA) for FoundationOneCDx to be used as a companion diagnostic for LYNPARZA (olaparib), which was also approved today in the U.S. for adult patients with deleterious or suspected deleterious germline or somatic homologous recombination repair (HRR) gene-mutated metastatic castration-resistant prostate cancer (mCRPC) who have progressed following prior treatment with enzalutamide or abiraterone. FoundationOne CDx is the only FDA-approved comprehensive genomic profiling (CGP) test for all solid tumors that incorporates multiple companion diagnostic claims.

Prostate cancer is the second most common cancer in men; 1 in 9 will be diagnosed during their lifetime.1 mCRPC occurs when prostate cancer grows and spreads to other parts of the body despite the use of androgen-deprivation therapy to block the action of male sex hormones.2 Because there have previously been limited treatment options for this specific disease area, there is generally a high mortality rate.

This therapy and companion diagnostic approval underscores the value of comprehensive genomic profiling in advanced cancer patients as it validates our ability to identify alterations in the 14 HRR pathway genes within FoundationOne CDxs 324 gene panel that indicate a patient may be eligible for treatment with Lynparza, a process not possible through single gene or hot spot testing, said Brian Alexander, M.D., M.P.H., chief medical officer at Foundation Medicine. This is an important advancement for patients with HRR-mutated metastatic castration-resistant prostate cancer, as there have previously been limited treatment options available for this specific condition.

FoundationOne CDx is the first FDA-approved broad companion diagnostic that is clinically and analytically validated for solid tumors. FoundationOne CDx is currently approved as a companion diagnostic for more than 20 targeted therapies.

LYNPARZA was approved based on the PROfound study, which was supported by Foundation Medicine and was the first phase III biomarker-selected study using a molecularly targeted treatment in men with metastatic castration-resistant prostate cancer (mCRPC) to demonstrate improved outcomes. The PROfound trial is the largest prospective study to date performing central tissue testing for homologous recombination repair (HRR) gene mutations in mCRPC patients. The clinical trial assay (CTA) is an NGS assay based on FoundationOne CDx.

LYNPARZA is jointly developed and commercialized by AstraZeneca (LSE/STO/NYSE: AZN) and Merck & Co., Inc.

About FoundationOne CDx

FoundationOne CDx is a next-generation sequencing based in vitro diagnostic device for detection of substitutions, insertion and deletion alterations (indels), and copy number alterations (CNAs) in 324 genes and select gene rearrangements, as well as genomic signatures including microsatellite instability (MSI) and tumor mutational burden (TMB) using DNA isolated from formalin-fixed paraffin embedded (FFPE) tumor tissue specimens. FoundationOne CDx is for prescription use only and is intended as a companion diagnostic to identify patients who may benefit from treatment with certain targeted therapies in accordance with their approved therapeutic product labeling. Additionally, FoundationOne CDx is intended to provide tumor mutation profiling to be used by qualified health care professionals in accordance with professional guidelines in oncology for patients with solid malignant neoplasms. Use of the test does not guarantee a patient will be matched to a treatment. A negative result does not rule out the presence of an alteration. Some patients may require a biopsy. For a full list of targeted therapies for which FoundationOne CDx is indicated as a companion diagnostic, please visit http://www.foundationmedicine.com/genomic-testing/foundation-one-cdx.

About Foundation Medicine

Foundation Medicine is a molecular information company dedicated to a transformation in cancer care in which treatment is informed by a deep understanding of the genomic changes that contribute to each patient's unique cancer. The company offers a full suite of comprehensive genomic profiling assays to identify the molecular alterations in a patients cancer and match them with relevant targeted therapies, immunotherapies and clinical trials. Foundation Medicines molecular information platform aims to improve day-to-day care for patients by serving the needs of clinicians, academic researchers and drug developers to help advance the science of molecular medicine in cancer. For more information, please visit http://www.FoundationMedicine.com or follow Foundation Medicine on Twitter (@FoundationATCG).

Foundation Medicine and FoundationOne are registered trademarks of Foundation Medicine, Inc.

Source: Foundation Medicine

1American Cancer Society Key Statistics for Prostate Cancer: https://www.cancer.org/cancer/prostate-cancer/about/key-statistics.html 2 Cancer.Net. (2019). Treatment of metastatic castration-resistant prostate cancer. http://www.cancer.net/research-and-advocacy/asco-care-and-treatment-recommendations-patients/treatment-metastatic-castration-resistant-prostate-cancer

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Foundation Medicine Receives FDA Approval for FoundationOneCDx as the Companion Diagnostic for LYNPARZA to Identify Patients with HRR-Mutated...

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When some people become infected with the coronavirus, they only develop mild or undetectable cases of COVID-19. Others suffer severe symptoms, fighting to breathe on a ventilator for weeks, if they survive at all.

Despite a concerted global scientific effort, doctors still lack a clear picture of why this is.

Could genetic differences explain the differences we see in symptoms and severity of COVID-19?

To test this, we used computer models to analyze known genetic variation within the human immune system. The results of our modeling suggest that there are in fact differences in peoples DNA that could influence their ability to respond to a SARS-CoV-2 infection.

When a virus infects human cells, the body reacts by turning on what are essentially anti-virus alarm systems. These alarms identify viral invaders and tell the immune system to send cytotoxic T cells a type of white blood cell to destroy the infected cells and hopefully slow the infection.

But not all alarm systems are created equal. People have different versions of the same genes called alleles and some of these alleles are more sensitive to certain viruses or pathogens than others.

To test whether different alleles of this alarm system could explain some of the range in immune responses to SARS-CoV-2, we first retrieved a list of all the proteins that make up the coronavirus from an online database.

We then took that list and used existing computer algorithms to predict how well different versions of the anti-viral alarm system detected these coronavirus proteins.

Also read: Worlds most accurate antibody test has arrived. Or has it?

The part of the alarm system that we tested is called the human leukocyte antigen system, or HLA. Each person has multiple alleles of the genes that make up their HLA type. Each allele codes for a different HLA protein. These proteins are the sensors of the alarm system and find intruders by binding to various peptides chains of amino acids that make up parts of the coronavirus that are foreign to the body.

Once an HLA protein binds to a virus or piece of a virus, it transports the intruder to the cell surface. This marks the cell as infected and from there the immune system will kill the cell.

In general, the more peptides of a virus that a persons HLAs can detect, the stronger the immune response. Think of it like a more sensitive sensor of the alarm system.

The results of our modeling predict that some HLA types bind to a large number of the SARS-CoV-2 peptides while others bind to very few. That is to say, some sensors may be better tailored to SARS-CoV-2 than others. If true, the specific HLA alleles a person has would likely be a factor in how effective their immune response is to COVID-19.

Because our study only used a computer model to make these predictions, we decided to test the results using clinical information from the 2002-2004 SARS outbreak.

Whats next?

We found similarities in how effective alleles were at identifying SARS and SARS-CoV-2. If an HLA allele appeared to be bad at recognizing SARS-CoV-2, it was also bad at recognizing SARS. Our analysis predicted that one allele, called B46:01, is particularly bad with regards to both SARS-CoV-2 and SARS-CoV. Sure enough, previous studies showed that people with this allele tended to have more severe SARS infections and higher viral loads than people with other versions of the HLA gene.

Based on our study, we think variation in HLA genes is part of the explanation for the huge differences in infection severity in many COVID-19 patients. These differences in the HLA genes are probably not the only genetic factor that affects severity of COVID-19, but they may be a significant piece of the puzzle. It is important to further study how HLA types can clinically affect COVID-19 severity and to test these predictions using real cases. Understanding how variation in HLA types may affect the clinical course of COVID-19 could help identify individuals at higher risk from the disease.

To the best of our knowledge, this is the first study to evaluate the relationship between viral proteins across a wide range of HLA alleles. Currently, we know very little about the relationship between many other viruses and HLA type. In theory, we could repeat this analysis to better understand the genetic risks of many viruses that currently or could potentially infect humans.

Austin Nguyen, PhD Candidate in Computational Biology and Biomedical Engineering, Oregon Health & Science University; Abhinav Nellore, Assistant Professor of Biomedical Engineering & Surgery, Oregon Health & Science University, and Reid Thompson, Assistant Professor of Radiation Medicine, Oregon Health & Science University

This article is republished from The Conversation under a Creative Commons license. Read the original article.

Also read: After HCQ, its time for azithromycin and pneumonia drug combo to go under clinical trial

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Its in your genes Whether Covid lands you in hospital or not depends on your body - ThePrint

Gender differences exist in many health conditions, and COVID-19 is no different. It appears that with regards to the novel coronavirus, mens health is less robust.

This global phenomenon is particularly visible in some countries. In Thailand, males account for a massive 81% of COVID-19 related deaths, in England and Wales, its 61%.

What are the reasons for the considerable difference between the sexes? We spoke to Dr Anthony Kaveh, MD, physician anesthesiologist, and integrative medicine specialist.

Men are disproportionately affected by COVID-19 than women. From preliminary data, possible reasons include behavioural, baseline health, and genetic differences between men and women, says Dr Kaveh.

Lets look at what we know about COVID-19 infections among men and women. But first, a little about how and why the sexes are different.

Men and women have vastly different biological characteristics, that develop thanks to our chromosomes. A chromosome is a bundle of coiled DNA, found in the nucleus of almost every cell in the body. Humans have 23 pairs of chromosomes.

The sex chromosomes determine whether you develop as a male or female.

In humans, women have two larger X chromosomes (XX), whereas men have a single X chromosome and a much smaller Y chromosome (XY) that has relatively fewer gene copies.

When an embryo is developing in the womb, these chromosomes dictate the future sex of the baby.

One of the genes found on the Y chromosomes, the SRY gene, starts testicular development in an XY embryo. The testicles begin to make testosterone which directs the embryo to develop as a male.

In an XX embryo, there is no SRY gene, so instead, an ovary develops which makes female hormones.

This basic, biological variation between the sexes can affect COVID-19 infection rates.

Although essential for male health, testosterone levels are also linked to a range of medical conditions.

Men are five times more likely to suffer an aortic aneurysm and three times more likely to develop kidney stones. Men also tend to die at a younger age than women.

Oestrogen is a predominantly female hormone that provides protective effects from conditions, including heart disease. Men cannot benefit from its positive health effects, as they only produce low levels.

However, Dr Kaveh says that The immunologic effects of oestrogen in protecting against COVID-19 are theoretical and dont yet provide a mechanism to explain our observations.

Oestrogen is a predominantly female hormone that provides protective effects from conditions.

Testosterone could have a role to play in COVID-19 infection rates. High levels of testosterone can suppress an immune response. Researchers found that women and men with lower levels of testosterone had higher antibody responses to an influenza vaccine.

The X chromosome has about 900 genes, the Y chromosome, just 55. Women have a genetic advantage with two X chromosomes because if there is a mutation in one, the other gene provides a buffer.

Men have more sex-linked diseases such as the blood clotting disorder, haemophilia, and suffer from an increased rate of metabolic disorders. The protective XX effect explains why male death rates are frequently higher.

The female immune system is stronger.

The female immune system is stronger. Concerning COVID-19 infections, Dr Kaveh says Genetic factors are often considered, including the more active female immune system. While a more active immune system would make sense to protect against COVID-19, it would be expected to worsen the cytokine storm we observe in severe COVID-19 infection.

However, there is no evidence to support that cytokine storms, which are potentially lethal, excessive immune responses, are more common in women.

If more men are testing positive for COVD-19, could the simple reason be that more men are tested than women? In fact, it seems the opposite is true.

Within the context of our early statistics, women are tested more frequently than men, but men have more positive tests. This may reflect a male stoicism that leads to delayed care, says Dr Kaveh.

Men are not as likely as women to seek medical attention. The Centers for Disease Control and Prevention (CDC) reported that women were 33% more likely than men to visit a doctor, even excluding pregnancy-related visits.

Women are tested more frequently than men, but men have more positive tests.

It seems like the reason for higher infection and death statistics in men is not due to a bias in testing.

Obesity, diabetes, hypertension, and smoking are also predictors of COVID-19 hospitalisation, but the breakdown is difficult to correlate, said Dr Kaveh.

People of either sex are more likely to suffer from complications from coronavirus if they have certain pre-existing health conditions, or engage in behaviours such as smoking and excessive alcohol consumption.

These health conditions and behaviours tend to be more common in men, which could affect the imbalance that we see in COVID-19 infections.

The association between risk factors and infection rate are not yet fully understood. For example, hypertension is more common in men until menopause, at which point female rates quickly rise, explains Dr Kaveh. In this case, we should be seeing an increase in the COVID-19 infection rate for women who have reached menopausal age, yet this is not the case.

The association between risk factors and infection rate are not yet fully understood.

Obesity, a risk factor for diabetes, affects women more than men globally. However, diabetes is slightly more prevalent in men. These comorbid conditions dont fully explain the COVID-19 observations, and neither does smoking, says Dr Kaveh.

Smoking is a risk factor for all respiratory diseases and also of lung cancer which is another COVID-19 risk factor.

In China, about 50% of men smoke and only 2% of women. These figures could contribute to the high ratio of male deaths which are more than double the rate of female deaths.

These differences in smoking and death rates are not as extreme in other countries. Risky behaviour cannot fully explain sex bias in COVID-19 infections.

As yet, it seems like there is no definitive answer as to why more men are suffering severe COVID-19 infections. More research is needed.

We are still very early in our global epidemiological observations of COVID-19. More complete data in the coming months will hopefully provide more clues to explain our observations, concluded Dr Kaveh.

Last updated: 30-04-2020

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Coronavirus deaths: why are more men dying from COVID-19 than women? - Netdoctor

For the first time, people with arm amputations can experience sensations of touch in a mind-controlled arm prosthesis that they use in everyday life. A study in the New England Journal of Medicine reports on three Swedish patients who have lived, for several years, with this new technology one of the world's most integrated interfaces between human and machine.The advance is unique: the patients have used a mind-controlled prosthesis in their everyday life for up to seven years. For the last few years, they have also lived with a new function sensations of touch in the prosthetic hand. This is a new concept for artificial limbs, which are called neuromusculoskeletal prostheses as they are connected to the user's nerves, muscles, and skeleton.

The research was led by Max Ortiz-Catalan, Associate Professor at Chalmers University of Technology, in collaboration with Sahlgrenska University Hospital, University of Gothenburg, and Integrum AB, all in Gothenburg, Sweden. Researchers at Medical University of Vienna in Austria and the Massachusetts Institute of Technology in the USA were also involved.

"Our study shows that a prosthetic hand, attached to the bone and controlled by electrodes implanted in nerves and muscles, can operate much more precisely than conventional prosthetic hands. We further improved the use of the prosthesis by integrating tactile sensory feedback that the patients use to mediate how hard to grab or squeeze an object. Over time, the ability of the patients to discern smaller changes in the intensity of sensations has improved," says Ortiz-Catalan.

"The most important contribution of this study was to demonstrate that this new type of prosthesis is a clinically viable replacement for a lost arm. No matter how sophisticated a neural interface becomes, it can only deliver real benefit to patients if the connection between the patient and the prosthesis is safe and reliable in the long term. Our results are the product of many years of work, and now we can finally present the first bionic arm prosthesis that can be reliably controlled using implanted electrodes, while also conveying sensations to the user in everyday life", continues Ortiz-Catalan.

Since receiving their prostheses, the patients have used them daily in all their professional and personal activities.

The new concept of a neuromusculoskeletal prosthesis is unique in that it delivers several different features which have not been presented together in any other prosthetic technology in the world:

The newest part of the technology, the sensation of touch, is possible through stimulation of the nerves that used to be connected to the biological hand before the amputation. Force sensors located in the thumb of the prosthesis measure contact and pressure applied to an object while grasping. This information is transmitted to the patients' nerves leading to their brains. Patients can thus feel when they are touching an object, its characteristics, and how hard they are pressing it, which is crucial for imitating a biological hand."Currently, the sensors are not the obstacle for restoring sensation," says Ortiz-Catalan. "The challenge is creating neural interfaces that can seamlessly transmit large amounts of artificially collected information to the nervous system, in a way that the user can experience sensations naturally and effortlessly."

The implantation of this new technology took place at Sahlgrenska University Hospital, led by Professor Rickard Brnemark and Doctor Paolo Sassu. Over a million people worldwide suffer from limb loss, and the end goal for the research team, in collaboration with Integrum AB, is to develop a widely available product suitable for as many of these people as possible.

"Right now, patients in Sweden are participating in the clinical validation of this new prosthetic technology for arm amputation," says Ortiz-Catalan. "We expect this system to become available outside Sweden within a couple of years, and we are also making considerable progress with a similar technology for leg prostheses, which we plan to implant in a first patient later this year."

The prosthesis is mind-controlled, via the electrical muscle and nerve signals sent through the arm stump and captured by the electrodes. The signals are passed into the implant, which goes through the skin and connects to the prosthesis. The signals are then interpreted by an embedded control system developed by the researchers. The control system is small enough to fit inside the prosthesis and it processes the signals using sophisticated artificial intelligence algorithms, resulting in control signals for the prosthetic hand's movements.

The touch sensations arise from force sensors in the prosthetic thumb. The signals from the sensors are converted by the control system in the prosthesis into electrical signals which are sent to stimulate a nerve in the arm stump. The nerve leads to the brain, which then perceives the pressure levels against the hand.

The neuromusculoskeletal implant can connect to any commercially available arm prosthesis, allowing them to operate more effectively.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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What's Everyday Life Like With a Mind-controlled Prosthetic Arm? - Technology Networks

Induced pluripotent stem cells made to produce insulin and CRISPR, used to correct a genetic defect, cured Wolfram syndrome in mice.

Using induced pluripotent stem cells (iPSCs) produced from the skin of a patient with a rare, genetic form of insulin-dependent diabetes called Wolfram syndrome, researchers transformed the human stem cells into insulin-producing cells and used CRISPR-Cas9 to correct a genetic defect that had caused the syndrome. They then implanted the cells into lab mice and cured the unrelenting diabetes in those models.

The findings, from researchers at Washington University School of Medicine in St. Louis, US, suggest this CRISPR-Cas9 technique may hold promise as a treatment for diabetes, particularly the forms caused by a single gene mutation and it also may be useful one day in some patients with the more common forms of diabetes, such as type 1 and type 2.

This is the first time CRISPR has been used to fix a patients diabetes-causing genetic defect and successfully reverse diabetes, said co-senior investigator Dr Jeffrey Millman, an assistant professor of medicine and of biomedical engineering at Washington University. For this study, we used cells from a patient with Wolfram syndrome because, conceptually, we knew it would be easier to correct a defect caused by a single gene. But we see this as a stepping stone toward applying gene therapy to a broader population of patients with diabetes.

Wolfram syndrome is caused by mutations to a single gene, providing the researchers an opportunity to determine whether combining stem cell technology with CRISPR to correct the genetic error also might correct the diabetes caused by the mutation.

Researchers at Washington University School of Medicine in St. Louis have transformed stem cells into insulin-producing cells. They used the CRISPR gene-editing tool to correct a defect that caused a form of diabetes, and implanted the cells into mice to reverse diabetes in the animals. Shown is a microscopic image of insulin-secreting beta cells (insulin is green) that were made from stem cells produced from the skin of a patient with Wolfram syndrome [credit: Millman lab Washington University].

Millman and his colleagues had previously discovered how to convert human stem cells into pancreatic beta cells. When such cells encounter blood sugar, they secrete insulin. Recently, these researchers developed a new technique to more efficiently convert human stem cells into beta cells that are considerably better at controlling blood sugar.

In this study, they took the additional steps of deriving these cells from patients and using the CRISPR-Cas9 gene-editing tool on those cells to correct a mutation to the gene that causes Wolfram syndrome (WFS1). Then, the researchers compared the gene-edited cells to insulin-secreting beta cells from the same batch of stem cells that had not undergone editing with CRISPR.

In the test tube and in mice with a severe form of diabetes, the newly grown beta cells that were edited with CRISPR more efficiently secreted insulin in response to glucose. Diabetes disappeared in mice with the CRISPR-edited cells implanted beneath the skin and the animals blood sugar levels remained in normal range for the entire six months they were monitored. Animals receiving unedited beta cells remained diabetic. Although their newly implanted beta cells could produce insulin, it was not enough to reverse their diabetes.

We basically were able to use these cells to cure the problem, making normal beta cells by correcting this mutation, said co-senior investigator Dr Fumihiko Urano, the Samuel E. Schechter Professor of Medicine and a professor of pathology and immunology. Its a proof of concept demonstrating that correcting gene defects that cause or contribute to diabetes in this case, in the Wolfram syndrome gene we can make beta cells that more effectively control blood sugar. Its also possible that by correcting the genetic defects in these cells, we may correct other problems Wolfram syndrome patients experience, such as visual impairment and neurodegeneration.

Were excited about the fact that we were able to combine these two technologies growing beta cells from induced pluripotent stem cells and using CRISPR to correct genetic defects, Millman said. In fact, we found that corrected beta cells were indistinguishable from beta cells made from the stem cells of healthy people without diabetes.

Moving forward, the process of making beta cells from stem cells should get easier, the researchers said. For example, the scientists have developed less intrusive methods, making iPSCs from blood and they are working on developing stem cells from urine samples.

The study is published in Science Translational Medicine.

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Induced pluripotent stem cells and CRISPR reversed diabetes in mice - Drug Target Review

CLAIM

"this coronavirus genome contained sequences of another virus [] the HIV virus (AIDS virus)"

DETAILS

Inaccurate: Genomic analyses indicate that the virus has a natural origin, and was not engineered. The so-called unique protein sequence insertions found in the 2019 novel coronavirus can be found in many other organisms, not just HIV.

KEY TAKE AWAY

Genomic analyses of the novel coronavirus show that it was not engineered. In addition, the claim that its genome contains inserted HIV sequences is based on a now-withdrawn preprint of a study that contained significant flaws in design and execution. The so-called HIV insertions identified by the authors are in fact gene sequences that can also be found in many other organisms besides HIV.

REVIEW Numerous articles published in April 2020 report that Nobel laureate Luc Montagnier claimed that SARS-CoV-2 is a manipulated virus that was accidentally released from a laboratory in Wuhan, China and that Indian researchers have already tried to publish the results of the analyses that showed that this coronavirus genome contained sequences of another virus [] the HIV virus (AIDS virus). The claim that SARS-CoV-2 contains HIV insertions began circulating in January 2020, and was propagated by outlets such as Zero Hedge and Infowars. Health Feedback covered this claim in early February 2020, and found it to be inaccurate.

Firstly, genomic analysis of the novel coronavirus, published in Nature Medicine, has demonstrated that the virus is not the product of bioengineering, but is rather of natural origin[1]. The current most likely theory, based on what scientists know about viral evolution, is that the virus first emerged in pangolins or bats (or both) and later developed the ability to infect humans. This ability to infect human cells is conferred by the so-called spike (S) protein, which is located on the surface of the enveloping membrane of SARS-CoV-2.

After the 2003-2005 SARS outbreak, researchers identified a set of key amino acids within the S protein which give SARS-CoV-1 a super-affinity for the ACE2 target receptor located on the surface of human cells[2,3]. Surprisingly, the S protein of the current SARS-CoV-2 does not contain this optimal set of amino acids[1], yet is nonetheless able to bind ACE2 with a greater affinity than SARS-CoV-1[4]. This finding suggests that SARS-CoV-2 evolved independently and undermines the claim that it was manmade[1]. Indeed, the best engineering strategy would have been to harness the known and efficient amino acid sequences already described in SARS-CoV-1 order to produce a more optimal molecular design for SARS-CoV-2. The authors of the Nature Medicine study[1] concluded that Our analyses clearly show that SARS-CoV-2 is not a laboratory construct or a purposefully manipulated virus.

Secondly, the claim that SARS-CoV-2 contains HIV insertions is based on a preprint of a research study uploaded to bioRxiv on 2 February 2020. A preprint is a study in progress that has not been peer-reviewed by other scientists. The authors of the preprint, titled Uncanny similarity of unique inserts in the 2019-nCoV spike protein to HIV-1 gp120 and Gag, claimed to have found 4 insertions in the spike glycoprotein (S) which are unique to 2019-nCoV and are not present in other coronaviruses. The authors further asserted that all of [these inserts] have identity/similarity to amino acids residues in key structural proteins of HIV-1 [which] is unlikely to be fortuitous in nature.

The work was swiftly criticized by experts. In this Forbes article, Arinjay Banerjee, a postdoctoral fellow at McMaster University who has studied coronaviruses, said that:

The authors compared very short regions of proteins in the novel coronavirus and concluded that the small segments of proteins were similar to segments in HIV proteins. Comparing very short segments can often generate false positives and it is difficult to make these conclusions using small protein segments.

Researchers also took to Twitter to demonstrate this problem first-hand. Trevor Bedford, a faculty member at the Fred Hutchinson Cancer Research Center who studies viral evolution, re-analyzed the gene and protein sequences used by the authors and found that the so-called unique inserts appeared in many other organisms, including Cryptosporidium and Plasmodium malariae, which cause cryptosporidiosis and malaria, respectively.

Assistant professor at Stanford University Silvana Konermann also checked the authors findings and came to the same conclusion, calling the similarity spurious.

This has also been independently confirmed in another published analysis[5]. In other words, these sequences are not insertions, but are rather common sequences found in numerous other organisms such as bacteria and parasites. Therefore, the existence of these sequences in SARS-CoV-2 does not provide evidence of a link to HIV, nor that scientists purposely inserted HIV sequences into the SARS-CoV-2 genome.

In summary, genomic analysis of the virus indicates that it does not contain so-called HIV insertions and that it was not engineered in a lab. Evidence points to the virus having a natural origin.

The only thing accurate about these articles is that Nobel Prize winner and virologist Luc Montagnier did in fact make these claims. Although he holds impressive scientific credentials, his claims run contrary to credible scientific evidence. And despite having won the Nobel Prize in Physiology or Medicine in 2008 for his co-discovery of the link between HIV and AIDS, Montagnier now promotes widely discredited theories such as the pseudoscience of homeopathy and that autism is caused by bacteria that emit electromagnetic waves. Articles which repeat Montagniers claims without critically evaluating their veracity exhibit the common appeal to authority fallacy, in which something is assumed to be true simply because the person saying it is considered to be an expert, thereby misleading readers into believing that this theory is scientifically credible. This demonstrates the importance of verifying scientific claims with other experts in the same field, rather than simply taking such claims from a single expert at face value.

SCIENTISTS FEEDBACK [These comments come from an evaluation of a related claim.] Aaron T. Irving, Senior Research Fellow, Duke-NUS Medical School:Its easier to believe misinformation when it is mixed with truth. The region highlighted in the pre-print is indeed an insertion in nCoV-2019 relative to its bat ancestors and indeed it has high identity to the HIV gp120/gag. However, the authors chose to align only this small region and not do a basic check on whether there were other sequences which were also homologous (showing high degree of similarity/identity). As it turned out, the region is also homologous to many unrelated sequences. As such, the conclusions drawn from the data are no longer valid and there are many open-ended questions regarding this region highlighted. I see the authors themselves agree with this criticism by other scientists and have voluntarily withdrawn their preprint pending a much deeper investigation.

The author of this article by European Scientist also compared the genome sequences of SARS-CoV-2 and HIV using the Basic Local Alignment Search Tool (BLAST), developed by the U.S. National Institutes of Health, and found no significant similarity, explaining that In plain English, SARS-CoV-2 is not made of the bat coronavirus and small bits of the HIV virus. Readers who wish to verify the level of sequence identity between the two viruses for themselves are welcome to follow the steps listed in the article.

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Nobel laureate Luc Montagnier inaccurately claims that the novel coronavirus is man-made and contains genetic material from HIV - Health Feedback

The coronavirus is running rampant through senior residential facilities, especially nursing homes. One source: Hospitals.

Ten years ago, as the conveyor belt of medicine geared up, profiteering hospitals learned to discharge patients as rapidly as possible. Some patients went home, but to expedite the transition, the path of least resistance was sending them to nursing homes.

To make them more palatable, nursing homes were rebranded as skilled nursing facilities, and many further enhanced their name to post-acute rehab. Yet, the care and reputation did not change.

Fast-forward to our present crisis. The glitch: Hospitals do not have to reveal whether medical staff members have tested positive for COVID-19 and continually hide behind the guise of confidentiality and HIPAA, shunning voluntary self-reporting. (Legally true in California. Legislators, are you listening?)

The existing hospital administrative attitude of get em in and get em out could therefore have created a vicious cycle of discharged patients contaminating residents at nursing facilities.

Moms, dads, aunts, uncles, sisters, brothers, veterans, retired teachers and first responders have been some of those vulnerable victims.

The roots of the present problem lie in the past, but we must dig in the future to connect the dots.

Gene Uzawa Dorio, M.D., is a geriatric house-call physician who serves as president of the Los Angeles County Commission for Older Adults and Assemblyman to the California Senior Legislature. He has practiced in the Santa Clarita Valley for 32 years.

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Connecting the Hospital-Nursing Home Dots | Doctor's Diary with Dr. Gene Dorio - SCVNEWS.com

REDWOOD CITY, Calif., April 20, 2020 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced the appointment of Scott Whitcup, M.D. to the Companys Board of Directors.

We are delighted to welcomeScott to our board, said Patrick Machado, J.D., Board Chair of Adverum Biotechnologies. Scott's expertise and accomplished track record as an ophthalmologist and successful drug developer fit perfectly with Adverum's mission to substantially elevate the standard of clinical care for patients suffering from neovascular AMD and other vision-threatening indications. My colleagues and I all look forward to working with Scott to realize fully the significant potential benefits our technology offers patients at risk of losing their sight.

This is an exciting time to join Adverums Board as the company strategically executes on expanding its pipeline through its novel vector discovery and drug development expertise to commercialize gene therapies to treat patients with serious ocular and rare diseases, said Dr. Whitcup. The development progress of ADVM-022, including the promising clinical data demonstrated to date in the OPTIC trial, has been impressive. I look forward to partnering with the Board, and the Adverum management team, on further developing the pipeline of drug candidates and advancing ADVM-022 towards commercialization for patients with wet AMD and diabetic retinopathy.

Scott Whitcup, M.D. is the founder and chief executive officer of Akrivista and Whitecap Biosciences, two companies focused on developing new therapies in ophthalmology and dermatology. In addition, he is on the clinical faculty at the UCLA Stein Eye Institute. Previously, Dr. Whitcup was the executive vice president of research and development and chief scientific officer at Allergan, where he led the discovery, clinical development, and medical affairs organizations focused on therapeutic areas including ophthalmology, CNS, urology, dermatology, and medical aesthetics. Earlier at Allergan, he served as vice president and head, ophthalmology therapeutic area, where he secured regulatory approvals for Alphagan P, Lumigan, Restasis, and Ozurdex. Earlier in his career, Dr. Whitcup was the clinical director at the National Eye Institute at the National Institutes of Health (NIH). Dr. Whitcup earned a B.A. from Cornell University and an M.D. from Cornell University Medical College. He completed an internal medicine residency at UCLA and an ophthalmology residency at Harvard University at the Massachusetts Eye and Ear Infirmary.

Dr. Whitcup serves on the board of directors of Scilex Pharmaceuticals and Anivive Lifesciences.

About Adverum BiotechnologiesAdverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy company targeting unmet medical needs in serious ocular and rare diseases. Adverum is evaluating its novel gene therapy candidate, ADVM-022, as a one-time, intravitreal injection for the treatment of its lead indication, wet age-related macular degeneration. For more information, please visit http://www.adverum.com.

Investor and Media Inquiries:Investors:Myesha LacyAdverum Biotechnologies, Inc.mlacy@adverum.com1-650-304-3892

Media:Cherilyn Cecchini, M.D.LifeSci Communicationsccecchini@lifescicomms.com 1-646-876-5196

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Adverum Biotechnologies Appoints Ophthalmology Industry Veteran Scott Whitcup, M.D. to Board of Directors - GlobeNewswire

Just because you've watched Jurassic Park doesn't mean you know that gene editing is bad

COURTESY OF FLICKR

Gene editing is an important area for further research.

Gene editing is the future and we should embrace it. I dont mean a wholehearted approval of the technique but to recognize that its here. We must be thoughtful about its applications and aware of its potential.

Weve been gene editing all throughout history. Selectively breeding animals and crops to promote the traits that are desirable or helpful to us. But todays gene editing is much different.

We use CRISPR-Cas9, which can target specific gene sequences to edit, said Samantha Noll, a bioethicist with the Functional Genomics Initiative.

CRISPR, a gene editing tool taken from bacterial defenses against viruses, allows molecular biologists here at WSU to alter specific genes in big animals. Compared to CRISPR, selective breeding is crude and inaccurate, only using phenotypic traits, such as eye or hair color, as the roadmap for which animal to breed or not. Selective breeding attempts to manipulate the genome by prioritizing expressed traits whereas CRISPR allows the manipulation of the genome by access to the entire gene pool.

Charlie Powell, the public information officer for WSUs school of Veterinary Medicine, mentioned multiple ways this gene editing technique can be applied positively.

At any given moment here in the US there are a million pigs in transit. A certain percentage of those animals will develop upper respiratory diseases as a result of the stress, Powell said. If we could make those pigs resistant instead of vaccinating them then we have the possibility of limiting those losses in the industry. This involves adding back the wild-type genes that they originated with.

This suggests ethical solutions by way of medical intervention. Lingering just on livestock application, how much animal suffering could be eliminated by well-applied selective gene editing? Instead of injecting tons of antibiotics, genetic immunity may be the way to go.

Fostering the path to healthier and happier livestock could be inroads to alleviating human challenges such as hunger and poverty. Abundance of sustainable and ethically produced meats could ease food demand, and resilient healthy livestock could be a valuable investment for underprivileged individuals.

Being a land-grant university, WSU research is primarily aimed at helping the local community, hence the focus on big animals and local agriculture. One of the research programs seeks to knock out the genes responsible for horns in cattle. This avoids the painful horn removal process for the animals and prevents accidental injury between cattle, which cost time and money.

Though these are promising initiatives, we cant be short-sighted either. William Kabasenche, a bioethicist focusing on the therapeutic applications of CRISPR, described what he called off-target effects.

Its called pleiotropy, when one gene influences multiple phenotypes, Kabasenche said.

Phenotypes are just the expressed traits. The information for those traits is stored in the gene. Off-target effects occur when a gene has unaccounted phenotypes, meaning that the manipulation of that gene produced an unforeseen or undesirable change in a phenotype.

This is why we have to be very careful when gene editing. Yes, the potential is huge both for scientific discovery as well as the well-being of conscious entities but we must guard against a utopic vision of the technology. There are trade-offs involved. Changing one gene may produce the desired effects, but drastically impact an unrelated but necessary function.

This stresses the need for research and the role of ethics in research. We should all want this work to be done, but we cannot simply focus on positive outcomes and draw the conclusion that it justifies its good. We must also consider how these outcomes are achieved.

We must also consider the harmful potential of gene editing. How we choose to engage our resources, the decisions and norms we set in research will in some part determine how well apply this technology. These norms are being born at research institutions like WSU.

Its a promising start that WSU includes ethicists, educators and biologists to tackle these difficult issues.

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OPINION: Support gene researchers - The Daily Evergreen

A team of researchers led by Dr Hugo Bellen at Baylor College of Medicine, investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Childrens Hospital and also a Howard Hughes Medical Institute investigator, and lead author, Hyunglok Chung, postdoctoral fellow in the Bellen lab, have discovered that a mutation in a gene that causes Mitchell disease.

However, they also discovered that the gene was not inherited, but was in fact a new mutation.

A patient with neurological symptoms enrolled in the Undiagnosed Diseases Network (UDN), presenting with an unidentified late-onset neurodegenerative disorder.

Bellen said: On comparing the patients and his parents DNA, the team identified a mutation in the patient that resulted in a single amino acid substitution (N237S) in the ACOX1 protein. This change was seen only in the patient and was not present in either of his parents DNA, indicating that the patient had a de novo, or new, mutation on this gene

With the help of the online gene-matching tool GeneMatcher, we found two more patients who had the same new mutation in the ACOX1 gene.

All three patients, who ranged from 3 to 12 years old at the time of disease onset, had remarkably similar clinical features, including degeneration of peripheral nerves that caused a progressive loss of mobility and hearing. The three individuals had identical gene variants, a clear indication that ACOX1 dysfunction likely was the cause of the symptoms.

The finding that an ACOX1 mutation was linked to Mitchell disease initially baffled the researchers. The only known ACOX1-related disorder described in the medical literature at that time presented earlier in infancy with seizures, severe cognitive decline, neuro-inflammation and accumulation of very-long-chain-fatty acids in plasma and, more importantly, was caused by the lack of the ACOX1 protein none of which was true for these three patients.

The brain has large amounts of lipids, which are critical for the proper functioning of the nervous system. Abnormal breakdown of lipids in the brain and peripheral nervous system is associated with several neurodegenerative diseases, Bellen said.

The gene ACOX1 is involved in lipid breakdown. It produces an enzyme called Acyl-CoA oxidase 1 that initiates a series of reactions that break down very-long-chain-fatty acids in small intracellular organelles called peroxisomes.

The team used fruit flies to understand the problem, with Chung discovering that the ACOX1 protein is abundant and critical for the maintenance of glia, cells that support neurons.

To gain a better understanding of how ACOX1 variants affect the function of glia, they generated two mutant fly lines, the first one lacked both the copies of ACOX1 gene and the second, carried the substitution mutation (N237S) found in one of the ACOX1 genes in the Mitchell disease patients.

Chung said: Flies lacking ACOX1 mimicked the symptoms of ACOX1 deficiency in humans, including elevated levels of very-long-chain-fatty acids along with dramatic loss of glia and neurons and progressively impaired neuronal function. When we reduced the synthesis of very-long-chain-fatty acids in these flies by administering the drug bezafibrate, we observed significant improvement in lifespan, vision, motor coordination and neuronal function, implicating elevated levels of these lipids and their excessive accumulation in glia as an important contributor.

The researchers suggest that bezafibrate could offer a new therapeutic avenue for patients.

In contrast to the loss of ACOX1, the introduction of the single amino acid substitution (N237S) in ACOX1 gene resulted in a hyperactive ACOX1 protein.

Typically, breakdown of very-long-chain-fatty acids by the enzymatic action of ACOX1 produces small amounts of highly reactive oxygen species, but glial cells quickly neutralise them. However, in Mitchells disease, hyperactive ACOX1 produces copious amounts of toxic reactive oxygen species, leading to the destruction of glia and their neighbouring neurons.

The harmful effects due to hyperactive ACOX1 were potently reversed with the antioxidant N-acetyl cysteine amide (NACA). However, NACA did not suppress the lethality or toxic effects in flies that lacked ACOX1, a clear indication that the two diseases act via entirely different pathways and would need to be treated with two distinct therapeutic strategies.

Bellen said: This study is a prime example of how combining UDNs unique team science approach with power of fruit fly genetics is facilitating rapid and phenomenal progress in rare diseases research. We take on cases of patients with conditions never described before, uncover new diseases, and find definitive molecular diagnosis for them. We make significant progress in unravelling the causes of these novel diseases and rapidly identify and test promising new treatment options.

We have successfully identified more than 25 disease-causing genes within the past three years a task that typically takes many years.

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Mitchell disease: solving the medical mystery - Health Europa

Global Gene Editing ServiceMarket: Trends Estimates High Demand by 2027

The Gene Editing ServiceMarket 2020 report includes the market strategy, market orientation, expert opinion and knowledgeable information. The Gene Editing ServiceIndustry Report is an in-depth study analyzing the current state of the Gene Editing ServiceMarket. It provides a brief overview of the market focusing on definitions, classifications, product specifications, manufacturing processes, cost structures, market segmentation, end-use applications and industry chain analysis. The study on Gene Editing ServiceMarket provides analysis of market covering the industry trends, recent developments in the market and competitive landscape.

It takes into account the CAGR, value, volume, revenue, production, consumption, sales, manufacturing cost, prices, and other key factors related to the global Gene Editing Servicemarket. All findings and data on the global Gene Editing Servicemarket provided in the report are calculated, gathered, and verified using advanced and reliable primary and secondary research sources. The regional analysis offered in the report will help you to identify key opportunities of the global Gene Editing Servicemarket available in different regions and countries.

The final report will add the analysis of the Impact of Covid-19 in this report Gene Editing Serviceindustry.

Some of The Companies Competing in The Gene Editing ServiceMarket are: Caribou Biosciences, CRISPR Therapeutics, Merck KGaA, Editas Medicine, Thermo Fisher Scientific, Horizon Discovery, Genscript Biotech, GeneCopoeia, Integrated DNA Technologies, Eurofins Genomics, DNA 2.0 (ATUM), BBI Life Sciences, Genewiz, Gene Oracle, SBS Genetech, and Bio Basic

Get a Sample Copy of the[emailprotected] https://www.reportsandmarkets.com/sample-request/covid-19-impact-on-global-gene-editing-service-market-size-status-and-forecast-2020-2026?utm_source=primefeed&utm_medium=15

The report scrutinizes different business approaches and frameworks that pave the way for success in businesses. The report used Porters five techniques for analyzing the Gene Editing ServiceMarket; it also offers the examination of the global market. To make the report more potent and easy to understand, it consists of info graphics and diagrams. Furthermore, it has different policies and improvement plans which are presented in summary. It analyzes the technical barriers, other issues, and cost-effectiveness affecting the market.

GlobalGene Editing ServiceMarket Research Report 2020 carries in-depth case studies on the various countries which are involved in the Gene Editing Servicemarket. The report is segmented according to usage wherever applicable and the report offers all this information for all major countries and associations. It offers an analysis of the technical barriers, other issues, and cost-effectiveness affecting the market. Important contents analyzed and discussed in the report include market size, operation situation, and current & future development trends of the market, market segments, business development, and consumption tendencies. Moreover, the report includes the list of major companies/competitors and their competition data that helps the user to determine their current position in the market and take corrective measures to maintain or increase their share holds.

What questions does the Gene Editing Servicemarket report answer pertaining to the regional reach of the industry?

The report claims to split the regional scope of the Gene Editing Servicemarket into North America, Europe, Asia-Pacific, South America & Middle East and Africa. Which among these regions has been touted to amass the largest market share over the anticipated duration

How do the sales figures look at present how does the sales scenario look for the future?

Considering the present scenario, how much revenue will each region attain by the end of the forecast period?

How much is the market share that each of these regions has accumulated presently

How much is the growth rate that each topography will depict over the predicted timeline

A short overview of the Gene Editing Servicemarket scope:

Global market remuneration

Overall projected growth rate

Industry trends

Competitive scope

Product range

Application landscape

Supplier analysis

Marketing channel trends Now and later

Sales channel evaluation

Market Competition Trend

Market Concentration Rate

Reasons to Read this Report

This report provides pin-point analysis for changing competitive dynamics

It provides a forward looking perspective on different factors driving or restraining market growth

It provides a six-year forecast assessed on the basis of how the market is predicted to grow

It helps in understanding the key product segments and their future

It provides pin point analysis of changing competition dynamics and keeps you ahead of competitors

It helps in making informed business decisions by having complete insights of market and by making in-depth analysis of market segments

TABLE OF CONTENT:

Chapter 1:Gene Editing ServiceMarket Overview

Chapter 2: Global Economic Impact on Industry

Chapter 3:Gene Editing ServiceMarket Competition by Manufacturers

Chapter 4: Global Production, Revenue (Value) by Region

Chapter 5: Global Supply (Production), Consumption, Export, Import by Regions

Chapter 6: Global Production, Revenue (Value), Price Trend by Type

Chapter 7: Global Market Analysis by Application

Chapter 8: Manufacturing Cost Analysis

Chapter 9: Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10: Marketing Strategy Analysis, Distributors/Traders

Chapter 11: Gene Editing ServiceMarket Effect Factors Analysis

Chapter 12: GlobalGene Editing ServiceMarket Forecast to 2027

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Research on Gene Editing Service Market (impact of COVID-19) with Top Players: Caribou Biosciences, CRISPR Therapeutics, Merck KGaA, Editas Medicine,...

-Spinal cord injury (SCI) creates a complex microenvironment that is not conducive to repair; growth factors are in short supply, whereas factors that inhibit regeneration are plentiful. In a new report, researchers have developed a structural bridge material that simultaneously stimulates IL-10 and NT-3 expression using a single bi-cistronic vector to alter the microenvironment and enhance repair. The article is reported in Tissue Engineering, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. Click here to read the article for free on the Tissue Engineering website through May 17, 2020.

In Polycistronic Delivery of IL-10 and NT-3 Promotes Oligodendrocyte Myelination and Functional Recovery in a Mouse Spinal Cord Injury Model, Lonnie D. Shea, PhD, University of Michigan, and coauthors report the development of a new poly(lactide-co-glycolide) (PLG) bridge with an incorporated polycistronic IL-3/NT-3 lentiviral construct. This material was used to stimulate repair in a mouse SCI model. IL-10 was included to successfully stimulate a regenerative phenotype in recruited macrophages, while NT-3 was used to promote axonal survival and elongation. The combined expression was successful; axonal density and myelination were increased, and locomotor functional recovery in mice was improved.

Inflammation plays a vital role in tissue repair and regeneration, and the use of a PLG bridge to take advantage of the inflammatory response to promote SCI repair is an elegant way to take advantage of these natural processes to improve SCI healing, says Tissue Engineering Co-Editor-in-Chief Antonios G. Mikos, PhD, Louis Calder Professor at Rice University, Houston, TX.

About the Journal

Tissue Engineering is an authoritative peer-reviewed journal published monthly online and in print in three parts: Part A, the flagship journal published 24 times per year; Part B: Reviews, published bimonthly, and Part C: Methods, published 12 times per year. Led by Co-Editors-in-Chief Antonios G. Mikos, PhD, Louis Calder Professor at Rice University, Houston, TX, and John P. Fisher, PhD, Fischell Family Distinguished Professor & Department Chair, and Director of the NIH Center for Engineering Complex Tissues at the University of Maryland, the Journal brings together scientific and medical experts in the fields of biomedical engineering, material science, molecular and cellular biology, and genetic engineering. Leadership of Tissue Engineering Parts B (Reviews) and Part C (Methods) is provided by Katja Schenke-Layland, PhD, Eberhard Karls University, Tbingen, Heungsoo Shin, PhD, Hanyang University; and John A. Jansen, DDS, PhD, Radboud University, and Xiumei Wang, PhD, Tsinghua University respectively. Tissue Engineering is the official journal of the Tissue Engineering & Regenerative Medicine International Society (TERMIS). Complete tables of content and a sample issue may be viewed on the Tissue Engineering website.

About the Publisher

Mary Ann Liebert, Inc., publishers is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including Stem Cells and Development, Human Gene Therapy, and Advances in Wound Care. Its biotechnology trade magazine, GEN (Genetic Engineering & Biotechnology News), was the first in its field and is today the industrys most widely read publication worldwide. A complete list of the firms 90 journals, books, and newsmagazines is available on the e Mary Ann Liebert, Inc., publishers website.

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Co-delivery of IL-10 and NT-3 to Enhance Spinal Cord Injury Repair - Mirage News

BRISBANE, Calif.--(BUSINESS WIRE)--Sangamo Therapeutics Inc. (Nasdaq: SGMO), a genomic medicine company, today announced the appointment of D. Mark McClung as Executive Vice President and Chief Business Officer. Mr. McClung will oversee commercial strategic planning, alliance management and corporate and business development.

Mr. McClungs appointment is the latest in the evolution of Sangamos leadership implemented over the last three years as the Companys technology and research programs have advanced into a diversified pipeline of therapeutic product candidates in various stages of clinical development. During this period, Sangamo has also appointed executive vice presidents overseeing R&D, manufacturing, legal and finance.

Im excited to welcome Mark to Sangamo. With our first product candidate entering Phase 3 and our broad pipeline of proprietary and partnered programs advancing in development, we are increasingly focused on late stage development and commercialization strategies for genomic medicines. Mark has extensive experience leading commercial organizations in therapeutic areas where innovative products have disrupted standards of care, said Sandy Macrae, Sangamos CEO.

From 2015 through 2019, Mr. McClung was Vice President and General Manager of Global Oncology Commercial at Amgen, which he joined from Onyx Pharmaceuticals where he had served as Chief Commercial Officer. For two decades prior, Mr. McClung held roles of increasing responsibility at GlaxoSmithKline in marketing and sales, commercial operations, and general management in the United States and Europe, including as Vice President and Head of Global Commercial for GSK Oncology from 2009 2013.

Over the next decade, genomic medicines have the potential to transform the practice of health care across therapeutic areas from rare monogenic diseases to immunology and oncology, and even to highly prevalent neurological disorders such as Alzheimers disease and Parkinsons disease, Mr. McClung commented. With its deep scientific expertise, diverse technology platforms, broad pipeline and significant collaborations, Sangamo is well positioned for this new era, and Im thrilled to join the Company at this time.

Stephane Boissel, Executive Vice President of Corporate Strategy, will leave Sangamo at the end of July and eventually return to an entrepreneurial project. Mr. Boissel joined Sangamo in 2018 following the acquisition of TxCell (now Sangamo France), where he had served as CEO.

Stephanes impactful contributions to Sangamo will endure for many years. He has driven several remarkable deals to fruition, including most recently our transaction with Biogen, which is among the largest preclinical collaboration deals ever, Macrae said. It has been an enormous pleasure working with Stephane these last two years, and we wish him every success in the future.

About Sangamo Therapeutics

Sangamo Therapeutics is committed to translating ground-breaking science into genomic medicines with the potential to transform patients lives using gene therapy, ex vivo gene-edited cell therapy, and in vivo genome editing and gene regulation. For more information about Sangamo, visit http://www.sangamo.com.

Sangamo Forward Looking Statements

This press release contains forward-looking statements regarding Sangamo's current expectations. These forward-looking statements include, without limitation, statements relating to the potential to develop, obtain regulatory approvals for and commercialize immunology and oncology therapies, therapies to treat rare monogenic diseases, neurological diseases and other diseases and other therapies and the timing and availability of such therapies, the potential for Sangamo to receive upfront licensing fees and earn milestone payments and royalties under the Biogen and other collaborations and the timing of such fees, payments and royalties, Sangamos product pipeline, technology platforms and scientific expertise, Sangamos financial resources and expectations and other statements that are not historical fact. These statements are not guarantees of future performance and are subject to risks and uncertainties that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, risks and uncertainties related to: the research and development process; the regulatory approval process for product candidates; the commercialization of approved products; the potential for technological developments that obviate Sangamo's technologies; the potential for Biogen to breach or terminate the collaboration agreement; and the potential for Sangamo to fail to realize its expected benefits of the Biogen and other collaborations. There can be no assurance that Sangamo will earn any upfront licensing fees or milestone or royalty payments under the Biogen or other collaborations or obtain regulatory approvals for product candidates arising from these collaborations. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in Sangamo's operations and business environments. These risks and uncertainties are described more fully in Sangamo's filings with the U.S. Securities and Exchange Commission, including its most recent Annual Report on Form 10-K. Forward-looking statements contained in this announcement are made as of this date, and Sangamo undertakes no duty to update such information except as required under applicable law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200417005086/en/

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Sangamo Appoints D. Mark McClung as Executive Vice President and Chief Business Officer - BioSpace

Snoqualmie, WA, July 03, 2020 --(PR.com)-- Gene C. Rousseau of Snoqualmie, Washington has been recognized as a Professional of the Year for 2020 by Strathmores Whos Who Worldwide Edition for his outstanding achievements and contributions for over 41 years in the machinery field.

About Gene C. RousseauGene C. Rousseau is the owner of TNG Machinery LLC, a longtime salesman for multiple woodworking companies covering Washington State and the Panhandle of Idaho who offers integrated metric solutions for wood, plastics and stone. TNG Machinery provides services to one man shops as well as large corporations. Their technology solutions can increase production and improve bottom lines. With over 41 years experience, Mr. Rousseau offers multiple values and liaises with cabinet companies. He provides metric solutions and assists with metric evaluations. Mr. Rousseau also serves as an intermediary for buying and selling used and new equipment.

Born on May 14, 1957 in Seattle, Washington, Gene attended Highline Community College and the University of Washington. Throughout his career, Mr. Rousseau has sold to the wood, plastic, stone and aerospace industries and has driven over 4.5 million miles. He was awarded Dealer of the Year in 2006 and 2007.

Gene married his beautiful wife Nicole in 1997 and they have two children, Tanner and Sydney. In his spare time, he enjoys fishing and coaching football.

For further information, contact https://www.tngmachinery.com.

About Strathmores Whos Who WorldwideStrathmores Whos Who Worldwide highlights the professional lives of individuals from every significant field or industry including business, medicine, law, education, art, government and entertainment. Strathmores Whos Who Worldwide is both an online and hard cover publication where we provide our members current and pertinent business information. It is also a biographical information source for thousands of researchers, journalists, librarians and executive search firms throughout the world. Our goal is to ensure that our members receive all of the networking, exposure and recognition capabilities to potentially increase their business.

Contact Information:Strathmore WorldwideSusan Perrault516-677-9696Contact via Emailwww.strathmoreworldwide.comSyndi Reibman

Read the full story here: https://www.pr.com/press-release/815106

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Gene C. Rousseau Recognized as a Professional of the Year for 2020 by Strathmore's Who's Who Worldwide - Benzinga

LONDON--(BUSINESS WIRE)--Technavio has been monitoring the regenerative medicine market and it is poised to grow by USD 9.55 billion during 2020-2024, progressing at a CAGR of over 20% during the forecast period. The report offers an up-to-date analysis regarding the current market scenario, latest trends and drivers, and the overall market environment.

Technavio suggests three forecast scenarios (optimistic, probable, and pessimistic) considering the impact of COVID-19. Request for Technavio's latest reports on directly and indirectly impacted markets. Market estimates include pre- and post-COVID-19 impact on the Regenerative Medicine Market Download free sample report

The market is fragmented, and the degree of fragmentation will accelerate during the forecast period. Allergan Plc, Amgen Inc., Hitachi Chemical Co. Ltd., Integra LifeSciences Holdings Corp., Medtronic Plc, MiMedx Group Inc., Organogenesis Holdings Inc., Smith & Nephew Plc, Takeda Pharmaceutical Co. Ltd., and Zimmer Biomet Holdings Inc. are some of the major market participants. The increasing prevalence of chronic diseases will offer immense growth opportunities. To make the most of the opportunities, market vendors should focus more on the growth prospects in the fast-growing segments, while maintaining their positions in the slow-growing segments.

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The increasing prevalence of chronic diseases has been instrumental in driving the growth of the market. However, uncertainties in regulatory approval might hamper market growth.

Technavio's custom research reports offer detailed insights on the impact of COVID-19 at an industry level, a regional level, and subsequent supply chain operations. This customized report will also help clients keep up with new product launches in direct & indirect COVID-19 related markets, upcoming vaccines and pipeline analysis, and significant developments in vendor operations and government regulations. https://www.technavio.com/report/regenerative-medicine-market-industry-analysis

Regenerative Medicine Market 2020-2024: Segmentation

Regenerative Medicine Market is segmented as below:

To learn more about the global trends impacting the future of market research, download a free sample: https://www.technavio.com/talk-to-us?report=IRTNTR41171

Regenerative Medicine Market 2020-2024: Scope

Technavio presents a detailed picture of the market by the way of study, synthesis, and summation of data from multiple sources. Our regenerative medicine market report covers the following areas:

This study identifies the increasing number of clinical trials as one of the prime reasons driving the regenerative medicine market growth during the next few years.

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Regenerative Medicine Market 2020-2024: Key Highlights

Table of Contents:

Executive Summary

Market Landscape

Market Sizing

Five Forces Analysis

Market Segmentation by Technology

Customer Landscape

Geographic Landscape

Drivers, Challenges, and Trends

Vendor Landscape

Vendor Analysis

Appendix

About Us

Technavio is a leading global technology research and advisory company. Their research and analysis focus on emerging market trends and provides actionable insights to help businesses identify market opportunities and develop effective strategies to optimize their market positions. With over 500 specialized analysts, Technavios report library consists of more than 17,000 reports and counting, covering 800 technologies, spanning across 50 countries. Their client base consists of enterprises of all sizes, including more than 100 Fortune 500 companies. This growing client base relies on Technavios comprehensive coverage, extensive research, and actionable market insights to identify opportunities in existing and potential markets and assess their competitive positions within changing market scenarios.

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COVID-19 Impact and Recovery Analysis- Regenerative Medicine Market 2020-2024 | Increasing Prevalence of Chronic Diseases to Boost Growth | Technavio...

NEWARK, Del., May 19, 2020 /PRNewswire/ --QPS, a global contract research organization (CRO) that provides discovery, preclinical, and clinical drug development services, is reinforcing its focus on qualitative and quantitative bioanalysis of biotherapeutics. QPS announces an expanded and upgraded fleet of high-resolution mass spectrometers (HRMS), with the addition of three new TripleTOF HRMS systems for GLP quantitation, two in the Newark, Delaware facility and one in the Suzhou, China laboratory. As part of this expansion, QPS has hired Larry Mallis, Ph.D., Director of Bioanalysis, to lead the newly merged biotherapeutics and biomarkers Liquid ChromatographyMass Spectrometry (LC-MS) quantitation team in Delaware.

The QPS facility in Newark, Delaware, now has four high-resolution mass spectrometers: two 6600+, one 6600, and one 5600, all of which are being used for Good Laboratory Practice (GLP) quantitation and metabolite identification.

"QPS has been closely watching the trends in the market and we are committed to responding to the needs of our clients by dedicating four of the five TripleTOF Ultra-Performance Liquid ChromatographyHigh-Resolution Mass Spectrometry (UPLC-HRMS) systems to quantitation of oligonucleotides and intact proteins," said John Kolman, VP, Global Head of Translational Medicine, QPS LLC.

"This increase in capacity and capability comes at a pivotal moment for QPS in China, as we continue our two-decades-long global effort to support the pharma industry in developing antiviral therapeutics and/or vaccines. This has become a higher priority in view of the new public health concerns due to the novel coronavirus," said Yondong Zhu, VP, Head of Bioanalytical Services, QPS China.

Larry Mallis, Ph.D., leader of the newly formed team in Newark, Delaware, built his career in the pharmaceutical industry (BMS, Wyeth, and Merck), before moving into the CRO industry, most recently as Director of Bioanalytical Operations at Lovelace Biomedical Research Institute. This new group now has all the necessary LC-MS and other chromatographic technology for PK/PD bioanalysis to support clients in drug discovery and development of rare diseases. This group's expertise lies in the quantitation of oligonucleotides, peptides, intact proteins, and highly hydrophilic low-molecular-weight metabolite biomarkers by UPLC-HRMS, or by immunoaffinity UPLC-MS/MS (tandem mass spectrometry), or by hybridization-LC-fluorescence.

ABOUT QPS HOLDINGS, LLC

Since 1995, QPS has provided discovery, preclinical, and clinical drug development services. An award-winning leader focused on bioanalytics and clinical trials, QPS is known for proven quality standards, technical expertise, a flexible approach to research, client satisfaction, and turnkey laboratories and facilities. QPS has CLIA-certified and GLP-compliant laboratories ready to fast-track your novel coronavirus and COVID-19 RT-qPCR/QPCR and Serological Assays and vaccine development programs. For more information, visitwww.qps.comor email[emailprotected].

QPS CONTACT:

Name: Gabrielle Pastore Phone: 1-302-635-4290 Email: [emailprotected]

SOURCE QPS

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QPS Continues to Expand UPLC-HRMS Quantitation Capabilities to Support Gene Therapy and Protein Drug Development - PRNewswire

Related ResearchSarepta Therapeutics Inc., a leader in precision genetic medicine for rare diseases, plans to invest over $30 million to expand its Gene Therapy Center of Excellence in Columbus, Ohio. The project is expected to create 100 new jobs.

Pending state approvals, Sarepta plans to open its new center in an 85,000-square-foot facility at 3435 Stelzer Road in Columbus. All employees currently operating out of the Companys Dublin, Ohio, office will transition to the new facility over time. Armed with the most advanced science in genetic medicine, the new center will allow Sarepta to lead the way in making Columbus an epicenter of gene therapy research and development. In addition to the new building, the companys investment will be used toward R&D equipment.

Sarepta is emerging as the world leader in gene therapy to treat and transform lives otherwise diminished and stolen by rare genetic disease. We are confident that gene therapy will revolutionize genetic medicine, and we chose Ohio for our Gene Therapy Center of Excellence because we believe Columbus will become a hub for genetic medicine innovation, said Sarepta President & CEO Doug Ingram. Our new center will strengthen the position of Columbus as a gene therapy leader, building on the advances of our long-standing partner, Nationwide Childrens Hospital.

We are proud to be the home of Sareptas new Gene Therapy Center of Excellence and to support their continued innovation within a critical field in medicine, said Columbus Mayor Andrew J. Ginther. The companys investment in Columbus is a testament to why our city and region are positioned to become the most important place in the world for gene therapy development.

From medical breakthroughs to cutting-edge technology, the Columbus Region is home to one of the top healthcare industries in the country. With several renowned healthcare systems and companies, including OhioHealth, Nationwide Childrens Hospital and Cardinal Health, the Columbus Region employs more than 45,000 in the industry. The Regions large pool of IT talent, combined with its well-established healthcare sector, makes it a hotbed for innovation.

Sarepta is one of the nations fastest growing biotechnology companies focused on the development of precision genetic medicines, said JobsOhio President and CEO J.P. Nauseef. With Ohio talent, Sarepta will advance its R&D of neuromuscular and central nervous system diseases at its new gene therapy R&D Center in the Columbus Region.

Founded in 1980, Sarepta develops genetic medicines to treat rare diseases, and currently has two approved products for Duchenne muscular dystrophy (DMD) and a pipeline of more than 40 treatments in development. The companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing.

Headquartered in Cambridge, Massachusetts, Sarepta has locations across four continents, and its current Central Ohio research facility in Dublin employs approximately 40 full-time workers.

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Sarepta Therapeutics Expands Gene Therapy Center of Excellence in Columbus, Ohio - Area Development Online

By Jonny Lupsha, News Writer

According to the Fierce Biotech article, the mice who underwent the new gene therapy were injected with a gene that makes the protein follistatin, which in turn blocks a protein called myostatin. Myostatin regulates muscle growth. The therapy caused a significant buildup of muscle mass in the mice while also preventing obesity, the article said. The mice didnt just build muscle; they also nearly doubled their strength without exercising any more than they usually did. Despite being fed a high-fat diet, they had fewer metabolic issues and stronger hearts than did animals that did not receive the follistatin gene.

Scientists have been developing gene therapy for many years. It can change our bodies in many ways, and has potential serving as a treatment for cancer and muscular dystrophy.

The procedure that the mice underwent encapsulates what gene therapy isalthough scientists generally focus on people.

I define [gene therapy] as the addition of genes to humans for medical purposes, said Dr. David Sadava, Adjunct Professor of Cancer Cell Biology at the City of Hope Medical Center.

Dr. Sadava said gene therapy is based on four assumptions. First, whoever is doing the gene therapy has to know the gene thats involved in whichever problem needs to be treated. Second, they must have a normal, healthy copy of that gene available in the lab. Third, they must know where and when the gene is normally expressed. Finally, they have to be fairly certain what will happen when the gene is expressed normally.

Additionally, gene therapy must do several things in order to be considered successful.

First, gene therapy must get the gene into the appropriate cells, Dr. Sadava said. Second, gene therapy must get the gene expressed in those cells. Third, we have to get the gene integrated into the genome of the target cells so itll be there permanently. And fourth, you better not have any bad side effects to gene therapy, like any therapy in medicine.

According to Dr. Sadava, one kind of gene therapy is referred to as gene augmentation, and it comes into play when the functional product of a gene has been lost and no longer gets produced normally. By injecting a gene into someone, healthy copies of a protein product will be made and function restored.

We could hypothetically think of muscular dystrophy as a good target for gene therapy, he said. We know that muscles lack the protein dystrophinits an organizing proteinso well put in the good gene for good dystrophin.

Another kind of gene therapy is called target cell killing. Dr. Sadava said it uses a gene that either produces a poison that kills certain types of cells or it stimulates the immune system to do so. Target cell killing can be applied to cancer.

A gene is put into cancer cells that allows them to produce a protein that will make a toxic drug from a harmless chemical, Dr. Sadava said. So the idea is we inject a harmless chemical into the body, it goes all over the body and when it enters a tumor cell, its converted into a poison by the gene product of the gene that weve put in for gene therapy. So we might put in a gene that will cause a protein to be made that attracts killer T cells so the tumor will stick up its hand and say Come kill me now.'

Gene therapy is an exciting field in science and medicine with a lot of potential for humans. For now, it may seem like its just helping some overweight mice get a confidence boost, but the practical applications should shore up within our lifetime.

Dr. David Sadava contributed to this article. Dr. Sadava is Adjunct Professor of Cancer Cell Biology at the City of Hope Medical Center in Duarte, CA, and the Pritzker Family Foundation Professor of Biology, Emeritus, at The Claremont Colleges. Professor Sadava graduated from Carleton University with a B.S. with first-class honors in biology and chemistry. He earned a Ph.D. in Biology from the University of California, San Diego.

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Lab Mice Shed Fat and Build Muscle with Gene Therapy - The Great Courses Daily News

Dyne to evaluate therapies targeting genetic cause of FSHD under agreement with international research organization UMONS

Leading FSHD researcher Jeffrey Statland, M.D., appointed to Scientific Advisory Board

WALTHAM, Mass.--(BUSINESS WIRE)-- Dyne Therapeutics, a biotechnology company pioneering life-transforming therapies for patients with serious muscle diseases, today announced the acceleration of its programs in facioscapulohumeral muscular dystrophy (FSHD) through the exclusive licensing of technologies to target the disease, as well as the appointment of leading researcher Jeffrey Statland, M.D., to its Scientific Advisory Board. Dr. Statland is an associate professor of neurology in the Department of Neurology at the University of Kansas Medical Center. He is also the co-principal investigator for ReSolve (Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD), an ongoing observational study run by the FSHD Clinical Trial Research Network (CTRN) and supported by Dyne Therapeutics.

The intellectual property exclusively licensed by Dyne targets the gene DUX4, which is the genetic basis of FSHD, and was developed by Professor Alexandra Belayew and Dr. Frdrique Coppe at the University of Mons (UMONS) Molecular Biology Laboratory in Belgium. Dyne is advancing an FSHD program using this suite of DUX4-targeting technology in combination with its proprietary FORCETM platform.

FSHD is a rare, debilitating muscle disease for which there are no approved treatments. Dyne's proprietary FORCE platform enables targeted delivery of a therapeutic inside the muscle cells of FSHD patients, where it is expected to reduce aberrant expression of the DUX4 protein and halt the loss of muscle function that characterizes FSHD. People affected by FSHD experience muscle pain and progressive skeletal muscle loss throughout the body that significantly affects their strength, mobility and quality of life. The muscle weakness often starts in the face, making it difficult to smile, and may progress to the point where affected individuals become dependent upon the use of a wheelchair for mobility.

Dyne has made a commitment to alleviating the FSHD burden for affected individuals and families. Todays announcements represent an important step forward for Dyne as we pursue our mission of delivering life-transforming therapies for serious muscle diseases, said Joshua Brumm, president and chief executive officer of Dyne. Jeff brings comprehensive insights into FSHD, including cutting-edge research into molecular and neuroimaging biomarkers that may be useful in assessing disease progression in future clinical trials. The agreement with UMONS gives us exclusive access to intellectual property to target the genetic cause of FSHD and complements our own proprietary platform for precision delivery into muscle cells.

There is a critical need for therapies to treat FSHD, which takes a debilitating physical and emotional toll on affected individuals and families, said Dr. Statland. The Dyne team is advancing FSHD research with their support of the ReSolve study and I believe their FORCE platform holds great potential. I am delighted to join Dynes Scientific Advisory Board.

Dr. Statland is an associate professor of neurology at the University of Kansas Medical Center, with both clinical and research training in neuromuscular diseases. His primary research interest is in FSHD. In addition to co-leading the ReSolve Natural History Study, Dr. Statland, with his collaborators at the University of Rochester Medical Center, is developing a disease-specific patient-reported health inventory and molecular and neuroimaging biomarkers of disease activity for future FSHD clinical trials. Dr. Statland holds a B.A. from Sarah Lawrence College, an MFA from Emerson College and an M.D. from the University of Kansas School of Medicine. He completed residency training in the Department of Neurology at the University of Kansas Medical Center and also conducted a fellowship in experimental therapeutics of neurologic disorders in the Department of Neurology at the University of Rochester Medical Center. He is board certified by the American Board of Psychiatry and Neurology.

About Dyne Therapeutics

Dyne Therapeutics is pioneering life-transforming therapies for patients with serious muscle diseases. The companys FORCETM platform delivers oligonucleotides and other molecules to skeletal, cardiac and smooth muscle with unprecedented precision to restore muscle health. Dyne is advancing treatments for myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). Dyne was founded by Atlas Venture and is headquartered in Waltham, Mass. For more information, please visit http://www.dyne-tx.com, and follow us on Twitter, LinkedIn and Facebook.

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Dyne Therapeutics Accelerates Programs in Facioscapulohumeral Muscular Dystrophy (FSHD) with Exclusive Licensing of Technologies to Target Genetic...