Category Archives: Gene Medicine

UPDATED: Fri., April 14, 2017, 10:04 p.m.

Elson S. Floyd College of Medicine assistant research professor Jason Gerstner leads the research team studying fruit flies brains and sleep patterns. (Dan Pelle / The Spokesman-Review)

A research team led by a Washington State University biochemist could help scientists shed light on why we need to sleep, and why some people have an easier time resting than others.

Jason Gerstner, a research assistant professor at WSUs Elson S. Floyd College of Medicine, found that mutations in a sleep gene in the brain can cause humans, mice and fruit flies to have less restful sleep.

The results of the study were published in a peer-reviewed article in the journal Science Advances earlier this month.

It might sound odd, but scientists still arent sure why sleep is necessary. One theory is that sleep helps with memory formation and the brains growth and change: what scientists call neuroplasticity. Other theories maintain that sleep is restorative for the body and that it lowers metabolism, helping to conserve energy.

We still dont fully understand what biological function sleep is serving, Gerstner said. One of the ways we can get at answering that question is through examining neurobiological pathways.

Much of Gerstners research has focused on a particular gene, FABP7, thats been linked to sleep function. In previous research, Gerstner saw the genes expression cycles naturally during the day in mice, mirroring sleep-wake cycles.

For this study, Gerstners team looked at a sleep study of Japanese men, some of whom had a naturally occurring mutation in their FABP7 gene. Men with the mutation slept about as long as men without it, but their sleep was more fitful, with more bouts of time spent awake during the night.

Men with the mutation also reported more symptoms indicating clinical depression on an assessment, though neither group scored high enough to meet the criteria for depression. Gerstner said that suggests either the gene mutation itself or sleep disturbance might be linked to depression in some way.

There were no significant differences in health, age or sleepiness between the two groups of men.

The study showed similar restlessness in rats that had their FABP7 genes knocked out and in genetically engineered fruit flies with the same gene mutation. Because the mutation works the same way across species, its a promising finding for future research, and even for treatment of sleep disorders.

The FABP7 mutation causes the gene to create a different protein sequence. That affects which other proteins in the brain the sequences bind to, which in turn can influence a broad range of functions, like gene expression, inflammation and other brain functions.

The researchers also found the specific part of the brain, a star-shaped cell called an astrocyte, where FABP7 plays a role in sleep.

Previously, those cells were thought to be support cells for neurons, Gerstner said. Now, scientists are learning theyre important in their own right.

This is some of the earliest evidence that astrocytes really play a role in sleep, said Isaac Perron, a doctoral student in neurobiology at the University of Pennsylvania who worked with mice in the experiment.

Perrons interest is in sleep and nutrition. Because the proteins coded by FABP7 bind with fatty acids like omega-3s, he thinks the gene might be a link in showing how the fatty acids we eat can influence brain functions, including sleep.

Jerry Yin, a professor of genetics at the University of Wisconsin-Madison, who worked on the fruit fly portion of the research, said finding a common pathway like FABP7 helps people looking at medications or gene therapies target their treatments.

Knowing FABP7 works in astrocytes helps researchers tailor their focus, since those cells are where youre likely to have an effect manipulating this gene, Yin said.

Testing a therapy or medication is also easier because the FABP7 impact on sleep works in fruit flies and mice, both of which are commonly used in research.

Since weve narrowed down particular protein expressed within astrocytes, it underscores the importance of these cells in regulating complex behavior across species, Gerstner said.

Link:
WSU sleep researchers discover why some people may toss and turn more than others - The Spokesman-Review

Using the new gene-editing enzyme CRISPR-Cpf1, researchers at UT Southwestern Medical Center have successfully corrected Duchenne muscular dystrophy in human cells and mice in the lab.

The UT Southwestern group had previously used CRISPR-Cas9, the original gene-editing system, to correct the Duchenne defect in a mouse model of the disease and in human cells. In the current work, they used a new variation of the gene-editing system to repair the defect in both a mouse model and in human cells.

"We took patient-derived cells that had the most common mutation responsible for Duchenne muscular dystrophy and we corrected them in vitro to restore production of the missing dystrophin protein in the cells. This work provides us with a promising new tool in the CRISPR toolbox," said author Dr. Eric Olson, Chairman of Molecular Biology, Co-Director of the UT Southwestern Wellstone Muscular Dystrophy Cooperative Research Center, and Director of the Hamon Center for Regenerative Science and Medicine.

The research appears in the journal Science Advances.

CRISPR-Cpf1 differs from CRISPR-Cas9 in a number of key ways. Cpf1 is much smaller than the Cas9 enzyme, which makes it easier to package inside a virus and therefore easier to deliver to muscle cells.

It also recognizes a different sequence of DNA than Cas9 does, which provides greater flexibility in terms of use. "There will be some genes that may be difficult to edit with Cas9 but may be easier to modify with Cpf1, or vice versa. The two proteins have different biochemical properties and recognize different DNA sequences, so these properties create more options for gene-editing," said Dr. Olson, who holds the Pogue Distinguished Chair in Research on Cardiac Birth Defects, the Robert A. Welch Distinguished Chair in Science, and the Annie and Willie Nelson Professorship in Stem Cell Research.

"By either skipping a mutation region or precisely repairing a mutation in the gene, CRISPR-Cpf1-mediated genome editing not only corrects Duchenne muscular dystrophy mutations but also improves muscle contractility and strength," said co-author Dr. Rhonda Bassel-Duby, Professor of Molecular Biology and Associate Director of the Hamon Center for Regenerative Science and Medicine.

Duchenne muscular dystrophy is caused by a mutation to one of the longest genes in the body. When there is a DNA error in the dystrophin gene, the body doesn't make the protein dystrophin, which serves as a sort of shock absorber for the muscle fiber. Since there are numerous places in the dystrophin gene where a mutation can occur, flexibility for gene-editing treatment is crucial.

Duchenne occurs in about 1 in every 5,000 boys, according to the Centers for Disease Control and Prevention. Duchenne muscular dystrophy is a progressive disease affecting both muscle used for movement and heart muscle, with patients typically succumbing before age 30 due to heart failure.

"CRISPR-Cpf1 gene-editing can be applied to a vast number of mutations in the dystrophin gene. Our goal is to permanently correct the underlying genetic causes of this terrible disease, and this research brings us closer to realizing that end," Dr. Olson said.

"CRISPR-Cpf1 differs from CRISPR-Cas9 in a number of key ways, including being easier to deliver to muscle cells, said Yu Zhang, a graduate student in Dr. Olson's lab and the first author of this study.

Story Source:

Materials provided by UT Southwestern Medical Center. Note: Content may be edited for style and length.

See the rest here:
Gene-editing alternative corrects Duchenne muscular dystrophy - Science Daily

A recent study from Dr. Song on the topic of research progress on treatment of cancer with Compatibility of Traditional Chinese Medicine establishes that the targeted gene therapy can be effective.

Xiangtan, China (PRWEB) April 15, 2017

Cancer is one of the major life-threatening diseases that people often worry about. People suffering from cancer often undergo traditional treatments, such as surgery, chemotherapy and radiotherapy. However, such treatments could have harmful side effects.

Research from Dr. Song(Dr. Xinping Song) was recently conducted aimed at studying traditional Chinese medicine compatibility with respect to treating prostate cancer. The study reveals that the targeted gene therapy can be an effective way.

The study also points out the targeted gene therapy can be combined with other therapies for a more effective result. However, this therapy is also not mature enough to address all health issues related to the prostatic cancer. In such a scenario, Dr. Song's 3D Prostate Targeted Treatment emerges as an innovative treatment for prostate cancer. The research revealed that the therapy can be combined with local targeted injections. The technique makes the therapy effective and increases its killing effect on cancer cells.

Dr. Song's treatment is based on the direct injection technique of the traditional Chinese medicine system. Dr. Xinping Song acknowledges the findings of the research and also the anti-cancer extract compatibility of the traditional Chinese medicine. In this prostate cancer treatment, patients are given small targeted injections in the affected areas of the prostate to help eliminate causative pathogens and clear the blockage. The injections carry herbal extracts only.

Dr. Song believes that the traditional Chinese anticancer medicine can better interpose with the symptoms of cancer patients. Dr. Song's prostate cancer treatment that follows the principles of traditional Chinese medicine is a clinical breakthrough. This innovative treatment brings more advantages in patients and their family's lives.

At Dr. Song 3D Urology and Prostate Clinic, patients can undergo all types of prostate care and treatment, including the treatment for the prostate cancer. This natural treatment method saves the cost and also meets the patient's requirements. With a non-surgical and quality treatment, patients can gradually improve their health and get rid of their pain and sufferings. To know more about Dr. Song's 3D prostate treatment, one can visit the website http://www.prostatecancer.vip/.

About 3D Urology and Prostate Clinic

The 3D Urology and Prostate Clinic is a premier prostate treatment clinic. The clinic specializes in treating various types of prostate diseases and complications, such as prostatitis, enlarged prostate, benign prostatic hyperlasia (BPH), prostate cancer, seminal vesiculitis, epididymitis,cystitis, prostate blockage and calcification, and chronic pelvic pain syndrome (CPPS), etc. The clinic is a medical clinic, licensed and approved by the Ministry of Health of the People's Republic of China.

For Media Enquiry Contact Person: Alisa Wang Telephone: 86-186-7321-6429 WhatsApp: + 86 -186-73216429 Email: prostatecure3d@gmail.com Website: http://www.prostate-3dcure.com

For the original version on PRWeb visit: http://www.prweb.com/releases/Dr-Song/04/prweb14247467.htm

See the original post:
Recent advances in experiment and study of prostate cancer targeted therapy - Benzinga

Consensus Study

Background

With the conclusion of the International Summit on Human Gene Editing, the second component of the Human Gene-Editing Initiative began: a comprehensive study of the scientific underpinnings of human gene-editing technologies, their potential use in biomedical research and medicine -- including human germline editing -- and the clinical, ethical, legal, and social implications of their use.

A multidisciplinary committee of expertsbegan its information-gathering process at the summit, and over the past year performed its own independent and in-depth review of the science and policy of human gene editing by reviewing the literature and holding data-gathering meetings in the U.S. and abroad to solicit broad input from researchers, clinicians, policymakers, and the public. The committee also monitored for the latest scientific achievements of importance in this rapidly developing field.

While informed by the statement issued by the organizing committee for the international summit, the consensus study committee had broad discretion to arrive at its own findings and conclusions. The committee's report represents the official views of NAS and NAM.

Moreabout the study| Info onpublic meetings | Answers tofrequently asked questions

Human Genome Editing: Science, Ethics, and Governance

Genome editing is a powerful new tool for making precise alterations to an organisms genetic material. Recent scientific advances have made genome editing more efficient, precise, and flexible than ever before. These advances have spurred an explosion of interest from around the globe in the possible ways in which genome editing can improve human health. The speed at which these technologies are being developed and applied has led many policymakers and stakeholders to express concern about whether appropriate systems are in place to govern these technologies and how and when the public should be engaged in these decisions.

In Human Genome Editing: Science, Ethics, and Governance, a new report from the National Academy of Sciences and National Academy of Medicine, an expert committee considers important questions about the human application of genome editing including: Balancing potential benefits with unintended risks, governing the use of genome editing, incorporating societal values into clinical applications and policy decisions, and respecting the inevitable differences across nations and cultures that will shape how and whether to use these new technologies. The committee sets forth criteria that must be met before permitting clinical trials of heritable germline editing, provides conclusions on the crucial need for public education and engagement, and presents seven general principles for the governance of human genome editing.

A Report Highlights brief is available:

The following one-pagers are also available:

Watch the archived video webcast of the report release briefing, held Feb. 14, 2017.

See the original post here:
Consensus Study on Human Gene Editing - National-Academies.org

Gene editing, through a technology called CRISPR , can edit DNA almost as easily as a word processor edits text. Now that system is being used to diagnose the presence of cancer and to create on-the-spot tests for infections like Zika virus , according to a new report published in the journal Science from researchers at the Broad Institute of MIT and Harvard.

CRISPR-cas9, first described in 2012, gives scientists the genetic equivalent of a word processing cursor, allowing them to home in on specific parts of a genome to delete, insert, copy, cut or paste DNA at will. In the current paper, Feng Zhang , associate professor of biological engineering and brain and cognitive sciences at MIT and a member of the Broad Institute, and his colleagues modified the CRISPR system so that it recognized the products churned out by genes as well. They used CRISPR to recognize specific substances that bacteria and viruses make. Picking up even the slightest whisper of these products can alert doctors that an infection is active.

The system, dubbed SHERLOCK, could accurately distinguish between Zika virus and dengue virus from blood, urine and saliva samples. SHERLOCK could also provide the answer more quickly than current tests. Now, it takes several days for doctors to culture microbes like bacteria or viruses in order to confirm that they are present. SHERLOCK, says Zhang, could identify a bacteria or virus in 30 minutes to an hour.

MORE : CRISPR Technology Scientists on Their Gene Editing Tool

In Zhangs tests, SHERLOCK also provided a way to measure how much virus was there, giving doctors a better sense of how entrenched the infection might be.

SHERLOCK could also pick up mutations present in tumors from patients blood. Such blood-based tests for cancer, or liquid biopsies , are a promising area of cancer research, and having a CRISPR-based tool like SHERLOCK could speed their development.

Zhang anticipates that the technology could also be critical in the fight against antibiotic resistance; SHERLOCK could determine if a persons bacterial infection is already resistant to certain antibiotics, for example, which would help doctors to avoid those drugs. It could also pick up when a persons infection starts to become resistant to the drug so doctors can switch a patients antibiotic to better fight the bacterial infection.

MORE : 5 Common Myths About Antibiotic-Resistant Bacteria

This new CRISPR based platform has the potential to be developed as a point of care diagnostic that could be as easy to use as an at-home pregnancy test, says James Collins, member of the Broad and professor of engineering at MIT.

The CRISPR system just requires a kit designed to recognize the bacteria, virus or cancer mutation in question, and those are relatively cheap and accessible. Zhangs team predicts some tests could cost less than $1. That could make it a mainstay in lower-resource countries when infectious like Zika or even Ebola emerge, by helping people to get treatment sooner so that the breadth of the outbreak is contained.

We want to deploy this in an area where it can make a real difference, says Zhang.

See original here:
Here's Another Way CRISPR Is Changing Medicine - TIME

Since actress Angelina Jolie had a preventative double mastectomy based on foreboding genetic testing in 2013, a study of 2,000 cancer patients conducted by Stanford University found that roughly half of those who opted for a bilateral mastectomy had a gene variant that may have been harmless.

All of the patients in the study had been recently diagnosed with cancer and had been tested for the BRCA1 and BRCA2 genetic mutations prior to having a bilateral, or double, mastectomy.

The study found that half of the women who had a bilateral mastectomy after having the genetic testing had variants of uncertain significance, or VUS, which may not correspond to a higher cancer risk.

The genetic testing has become more common because it is less expensive than it used to be. But interpretations of the results can be complicated and confusing.

Between 25 and 50 percent of the breast cancer surgeons admitted to treating women with the VUS the same way they would treat women with known cancer-associated variants. This may have led less-experienced surgeons to recommend aggressive bilateral mastectomy when the involvement of a genetic counselor might have led to a different result.

In a review of the study on the Stanford Medicine News Center, Allison Kurian, MD, said, Our findings suggest a limited understanding among physicians and patients of the meaning of genetic testing results.

An earlier study, conducted in February, found that doctors often did not recommend genetic testing for breast cancer patients who were at high risk for carrying the BRCA1 or BRCA2 genes.

That study surveyed 2,502 women who had recently been diagnosed with breast cancer. Among them, 666 had received the genetic testing with 59% of that group testing positive for the genes. A quarter of that group did not have the testing done until after completing surgery. Women without private medical insurance were more likely to have delayed testing.

Our findings suggest that we are not maximizing the benefit of genetic testing for our patients with breast cancer because of barriers related to timeliness of testing and lack of expertise necessary to incorporate results into treatment decisions, said University of Michigan researcher Steven Katz, MD, MPH.

Originally posted here:
Stunning Allegation: Half of all mastectomy patients do NOT have cancer gene; Misreading data? - CrimeOnline

Scientists at the Centre for Addiction and Mental Health (CAMH) and Queen's University have identified 26 new genes linked to intellectual disability. Currently most patients with intellectual disability receive no molecular diagnosis, significantly affecting their health and shortening their lifespan, according to the researchers.

The study ("Mapping Autosomal Recessive Intellectual Disability: Combined Microarray and Exome Sequencing Identifies 26 Novel Candidate Genes in 192 Consanguineous Families"),published inMolecular Psychiatry, has implications for the diagnosis and clinical care of those affected, and also adds to the growing knowledge of brain development and functioning. It may eventually lead to personalized treatments for affected individuals. Interestingly, some of the genes identified are thought to be connected with autism spectrum disorders, notes John Vincent, Ph.D.,team leader and senior scientist who heads the MiND (Molecular Neuropsychiatry and Development) Laboratory in theCampbell Family Mental Health Research Instituteat CAMH.

"This is the largest study of its kind on intellectual disability to come out of North America," he adds.

More than one in 100 children worldwide are affected by intellectual disability, which is characterized by significant limitations in learning that also affect their day-to-day lives. Frequently, intellectual disability also accompanies symptoms of autism spectrum disorders, and many genes have been found to be shared by the two illnesses.

The study involved 192 families from Pakistan and Iran with more than one affected family member. Intellectual disability is frequently caused by recessive genes, meaning that an affected child gets a defective copy of the gene from each parent.

The families in the study all had a history of marriage among relatives, which occurs quite commonly in communities in South Asia, the Middle East, and Africa. Studying families with this background, and multiple affected individuals, can enable researchers to identify disease genes that would otherwise remain hidden.

The Canadian research team pinpointed mutations related to intellectual disability in half of these 192 families, in 72 different genes. The identification of 26 new genes adds to 11 new genes that the team had previously linked to intellectual disability.

One immediate implication of the study is to prevent future cases of intellectual disability, the researchers say. Unaffected family members and relatives could be genetically screened to see if they carry these mutations and provided with counselling on the risks of "within-family" marriages.

A broader goal is to develop diagnostic screening tools that are also relevant to populations in which "within-family" marriages are rare, such as Canada, U.S., Japan, China, and Europe. Ultimately, this information would be used to plan more personalized treatment.

While 26 genes may seem a substantial number, there are likely hundreds of genes that, when defective, may lead to intellectual disability. "The strategy we have used speeds up the process of identifying disease genes and of enabling diagnostic labs to deliver more accurate information for clinicians and families," explains Dr. Vincent.

This strategy involves various genetic techniques, including microarray genotyping and whole-exome sequencing, and studying families with a history of marriage among relatives.

"There's an opportunity now to further explore the functioning and biological pathways of these genes, and to help complete the picture of how the central nervous system works," continued Dr. Vincent. "Knowing the genes involved is a big step forward, but understanding how they function is also crucial before we can start planning treatments or even cures."

Read more:
Gene Findings Advance Precision Medicine for Intellectual Disability - Genetic Engineering & Biotechnology News

April 13, 2017 by Fred Gould Credit: North Carolina State University

Providing blanket approval or condemnation of all genetically engineered (GE) crops oversimplifies a complex issue and ignores the continued need for scrutiny, risk assessment and debate among various stakeholders including scientists, farmers and the general public.

That's the main message of a letter, "Elevating the Conversation About GE Crops," published this week in Nature Biotechnology. It responds to a December 2016 critique of last May's U.S. National Academies of Sciences, Engineering, and Medicine report on genetic engineering.

Fred Gould, William Neal Reynolds Professor of Agriculture at North Carolina State University and co-director of the university's Genetic Engineering and Society Center, chaired the National Academies committee responsible for the report, Genetically Engineered Crops: Experiences and Prospects, which aimed to "assess the evidence for purported negative effects of GE crops and their accompanying technologies" and to "assess the evidence for purported benefits of GE crops and their accompanying technologies." Gould is also the corresponding author of the letter, which represents the views of the 20-person committee that produced the report.

"It is not surprising that our detailed conclusions, which are often crop-, trait- and context-specific, do not sit well with those who want a universal thumbs-up or thumbs-down to GE crops," Gould said. "It is a nuanced report because the issue is not black and white. Maybe the greatest value of the report is pointing out that there is no straightforward answer."

The scathing critique, published in Nature Biotechnology late last year, called for blanket approval of GE crops and derided the report for a number of what it called "inaccuracies" and "omissions."

"This unwillingness to overtly back GE crops, and the report's efforts to give credence to alternative viewpoints rather like the media's obsession with giving two sides of an argument equal play, irrespective of which view is supported by the evidence is puzzling," the critique stated.

This specific critique is, in effect, an uninformed indictment of the National Academies' process, Gould says.

He points to the letter's description of the painstaking way the report was produced.

"For the three report chapters concerning currently commercialized GE crops, our report includes over 900 references," the letter states. "Once our committee developed a full draft of the report, it was sent to 26 reviewers with diverse expertise and perspectives (these reviewers were anonymous to the committee until they were acknowledged in the final report). Each of the 918 comments and criticisms in the reviews had to be specifically addressed by the committee to the satisfaction of a National Academies' independent review board before the report could move forward for the Academies' approval. With all this input and review, this report clearly represented more than the opinions of the 20 committee members."

The critique also accused the report of paying too much attention to non-experts the general public.

"Science is not democratic," the critique asserted. "The citizenry do not get to vote on whether a whale is a mammal or a fish, the temperature at which water boils, or whether the number 'pi' should be rounded off. There is no such public consultation with respect to the introduction of a new kind of flu vaccine or of new techniques of cardiovascular surgery."

Gould and the National Academies committee counter the criticism with a quote from a speech given in 1999 by former Agriculture Secretary Dan Glickman: " with all that biotechnology has to offer, it is nothing if it's not accepted. This boils down to a matter of trust. Trust in the science behind the process, but particularly trust in the regulatory process that ensures thorough review including complete and open public involvement."

"Secretary Glickman reminds us here that governing GE technology is more than just regulation," Gould said. "Public input is critical in order to build trust. We highlight this important advice in both the report's preface and the letter. It really gets to the heart of the issue."

Gould says that rather than simplistic approval or condemnation of GE crops, the report found that the social and economic effects of GE crops depended on whether the GE trait and the genetics of the cultivar it was put into matched the needs of the farmers and the farm environment.

The report also, in its discussion of regulating GE crops, concluded that it was not how a genetic change was made or even the amount of DNA that was altered that should be the focus of regulation. In the end, the report asserts, it is the plant characteristics that should be regulated. The committee concluded that these criteria applied as much to what are considered conventionally bred plants as to GE plants.

Gould adds that constituencies both for and against GE crops have weighed in on the report, making diametrically opposite claims about the report's findings. Some even suggested that there was no need for a report of this type.

"We welcome all public input into the report because these discussions are healthy and helpful," Gould said.

Explore further: Assessing the positive and negative claims about genetically engineered crops

More information: Fred Gould et al. Elevating the conversation about GE crops, Nature Biotechnology (2017). DOI: 10.1038/nbt.3841

Genetic engineering in general, and genetically engineered (GE) crops in particular, stir strong feelings from both critics and supporters. The National Academies of Sciences, Engineering, and Medicine have just released ...

Genetically manipulated food remains generally safe for humans and the environment, a high-powered science advisory board declared in a report Tuesday.

An extensive study by the National Academies of Sciences, Engineering, and Medicine has found that new technologies in genetic engineering and conventional breeding are blurring the once clear distinctions between these two ...

Genetically engineered (GE) crops are no different from conventional crops in terms of their risks to human health and the environment, according to a report published in May 2016 by the U.S. National Academies of Sciences, ...

Releasing genetically-modified mosquitoes into the wild to fight malaria, Zika or other insect-borne diseases is premature and could have unintended consequences, researchers said in a new report.

A profusion of biotechnology products is expected over the next five to 10 years, and the number and diversity of new products has the potential to overwhelm the U.S. regulatory system, says a new report from the National ...

People's ability to make random choices or mimic a random process, such as coming up with hypothetical results for a series of coin flips, peaks around age 25, according to a study published in PLOS Computational Biology.

Imagine that the way flies and butterflies drink nectar and other fluids can be imitated for use in medicine, potentially to deliver life-saving drugs to the bodyand also how this method can save their own lives in times ...

A team of scientists from the Broad Institute of MIT and Harvard, the McGovern Institute for Brain Research at MIT, the Institute for Medical Engineering & Science at MIT, and the Wyss Institute for Biologically Inspired ...

The bacterial flagellum is one of nature's smallest motors, rotating at up to 60,000 revolutions per minute. To function properly and propel the bacterium, the flagellum requires all of its components to fit together to exacting ...

Hunting is a major threat to wildlife particularly in tropical regions, but a systematic, large-scale estimate of hunting-induced declines of animal numbers has been lacking. A study published in Science on April 14 fills ...

There are many processes that take place in cells that are essential for life. Two of these, transcription and translation, allow the genetic information stored in DNA to be deciphered into the proteins that form all living ...

Adjust slider to filter visible comments by rank

Display comments: newest first

Among other things, many of the objections about genetically engineered foods come from the realization that, from the U.S.S. Maine, to claims of not flying spy planes over Russia, to the president turning the Oval Office into a cat house, to claiming there was mass production of banned weapons systems in Iraq, the government always lies, and the purpose is money or power of both. Saying "non-experts" don't get a vote ignores the fact that it was "non-experts" who discovered many things, including, for example, inoculation. It ignores the fact that, before any study was begun, everyone was a non-expert, and, if non-experts are not to be trusted, then no system of facts can be believed. It also ignores the fact that, in places like court trials, you can find "experts" who'll say anything you want.

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Read the rest here:
Committee responds to critique of gene engineering report - Phys.Org

MORRISTOWN, N.J., April 13, 2017 /PRNewswire/ --Significant knowledge gaps about gene therapies persist, despite surging interest and accelerating clinical development, according to new research presented by SmithSolve, a strategic communications agency specializing in rare and genetic disease,this week during the 2017 Gene Therapy for Rare Diseases conference hosted by the New York Academy of Sciences. The findings were summarized in a poster titled, Enhancing Awareness and Understanding of Gene Therapy among Rare Disease Communities: A Research-Driven Roadmap.

Researchers conducted a multifaceted study that included an analysis of peer-reviewed literature, a series of industry expert interviews, a patient and caregiver survey, and a one-year audit of social and traditional media coverage and conversation about gene therapy. Among the more notable findings:

"The gene therapy revolution is well underway, bringing with it a potentially massive shift in how we treat disease," said lead researcher Chris Smith, president and chief executive officer of SmithSolve. "Yet, despite the promise of gene therapy, our research identified a clear need for improved education and outlines a roadmap that all stakeholders can use to improve communication about this incredibly complex area of medicine."

Smith listed several opportunities to help bridge the informational gap and promote enhanced understanding of gene therapies. These include securing alignment among all stakeholders, establishing a common nomenclature, and fostering realistic expectations.

"Across dozens of rare genetic diseases, we are learning that early intervention leads to improved outcomes," noted Maria Escolar, MD, MS, director of the Program for the Study of Neurodevelopment in Rare Disorders at Children's Hospital of Pittsburgh, and associate professor of Pediatrics at the University of Pittsburgh School of Medicine. "This finding provides strong motivation for patients, advocates, physicians, research institutions, and industry to join together to improve awareness of gene therapies, so understanding keeps pace with research."

"We hope our research spurs the kind of dialogue and collaboration we recommend, and we look forward to facilitating these efforts," added co-author Patti Engel, RN, BSN, of Engage Health in Eagan, Minn.

The authors initiated this research to address the needs of rare disease patient communities and to fulfill Patient Advocacy Certificate Training (PACT) requirements of the Professional Patient Advocates in Life Sciences (PPALS). To learn more about the survey findings, visit http://smithsolve.com/results/ready-for-a-revolution/.

About SmithSolveSmithSolve, based in Morristown, N.J., is a strategic communications firm exclusively serving the healthcare industry. The company specializes in the rare disease and orphan drug sectors and helps established as well as early-stage biopharmaceutical companies tell their corporate narratives and translate complex science across multiple stakeholders. Its hands-on, senior-level team provides a broad range of communications services spanning product and pipeline news, patient advocacy, media relations, corporate reputation, issues management, digital design, and more. Established in 2006, SmithSolve partners with an established network of independent public relations agencies to design and execute programs worldwide. SmithSolve is a founding member of the Rare Collective, a group of seasoned experts in orphan drug development (www.rarecollective.org). For more information, visit http://www.smithsolve.com.

Read More

About Engage Health, Inc.Engage Health, based in Eagan, Minn., is an experienced marketdevelopmentfirm serving the pharmaceutical, biotechnology, and medical device sectors inthe commercialization of specialty medical products. The staff at Engage Health brings over 20 years of experience in understanding and developing rare disease markets.From market quantification, patient and caregiver identification/mapping, to critical market research, Engage helps clients make the right decisions in development to maximize the commercial success of rare disease and specialty products. Engage is a founding member of the Rare Collective, a group of seasoned experts in orphan drug development (www.rarecollective.org). For more information, visit http://www.engagehealth.com.

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/research-reveals-lingering-misconceptions-about-gene-therapy-as-promising-new-product-candidates-advance-300439670.html

See more here:
Research Reveals Lingering Misconceptions about Gene Therapy as Promising New Product Candidates Advance - Yahoo Finance

Gene therapy and genetic engineering are two closely related technologies that involve altering the genetic material of organisms. The distinction between the two is based on purpose. Gene therapy seeks to alter genes to correct genetic defects and thus prevent or cure genetic diseases. Genetic engineering aims to modify the genes to enhance the capabilities of the organism beyond what is normal.

Ethical controversy surrounds possible use of the both of these technologies in plants, nonhuman animals, and humans. Particularly with genetic engineering, for instance, one wonders whether it would be proper to tinker with human genes to make people able to outperform the greatest Olympic athletes or much smarter than Einstein.

We use the phrase genetic engineering more narrowly for the kind of alteration that aims at enhancement rather than therapy. We use the term gene therapy for efforts to bring people up to normalcy and genetic engineering or enhancement genetic engineering for efforts to enhancement peoples capabilities beyond normalcy.

There are several options for what kind of change to make to the gene. DNA in the gene could be replaced by other DNA from outside (called homologous replacement). Or the gene could be forced to mutate (change structure selective reverse mutation.) Or a gene could just be added. Or one could use a chemical to simply turn off a gene and prevent it from acting.

There are also several options for how to spread the genetic change to all the cells that need to be changed. If the altered cell is a reproductive cell, then a few such cells could be changed and the change would reach the other somatic cells as those somatic cells were created as the organism develops. But if the change were made to a somatic cell, changing all the other relevant somatic cells individually like the first would be impractical due to the sheer number of such cells. The cells of a major organ such as the heart or liver are too numerous to change one-by-one. Instead, to reach such somatic cells a common approach is to use a carrier, or vector, which is a molecule or organism. A virus, for example, could be used as a vector. The virus would be an innocuous one or changed so as not to cause disease. It would be injected with the genetic material and then as it reproduces and infects the target cells it would introduce the new genetic material. It would need to be a very specific virus that would infect heart cells, for instance, without infecting and changing all the other cells of the body. Fat particles and chemicals have also been used as vectors because they can penetrate the cell membrane and move into the cell nucleus with the new genetic material.

Genetic engineering to enhance organisms has already been used extensively in agriculture, primarily in genetically modified (GM) crops (also known as GMO --genetically modified organisms). For example, crops and stock animals have been engineered so they are resistant to herbicides and pesticides, which means farmers can then use those chemicals to control weeds and insects on those crops without risking harming those plants. In the future genetic enhancement could be used to create crops with greater yields of nutritional value and selective breeding of farm stock, race horses, and show animals.

Genetically engineered bacteria and other microorganisms are currently used to produce human insulin, human growth hormone, a protein used in blood clotting, and other pharmaceuticals, and the number of such compounds could increase in the future.

Enhancing humans is still in the future, but the basic argument in favor of doing so is that it could make life better in significant ways by enhancing certain characteristics of people. We value intelligence, beauty, strength, endurance, and certain personality characteristics and behavioral tendencies, and if these traits were found to be due to a genetic component we could enhance people by giving them such features. Advocates of genetic engineering point out that many people try to improve themselves in these ways already by diet, exercise, education, cosmetics, and even plastic surgery. People try to do these things for themselves, and parents try to provide these things for their children. If exercising to improve strength, agility, and overall fitness is a worthwhile goal, and if someone is praised for pursuing education to increase their mental capabilities, then why would it not be worthwhile to accomplish this through genetics?

Advocates of genetic engineering also see enhancement as a matter of basic reproductive freedom. We already feel free to pick a mate partly on the basis of the possibility of providing desirable children. We think nothing is wrong with choosing a mate whom we hope might provide smart, attractive kids over some other mate who would provide less desirable children. Choosing a mate for the type of kids one might get is a matter of basic reproductive freedom and we have the freedom to pick the best genes we can for our children. Why, the argument goes, should we have less freedom to give our children the best genes we can through genetic enhancement?

Those who advocate making significant modification of humans through technology such as genetic engineering are sometimes called transhumanists.

The danger objection points out that a few recent attempts at gene therapy in clinical trials have made headlines because of the tragic deaths of some of the people participating in the trials. It is not fully known to what extent this was due to the gene therapy itself, as opposed to pre-existing conditions or improper research techniques, but in the light of such events some critics have called for a stop to gene therapy until more is known. We just do not know enough about how gene therapy works and what could go wrong. Specific worries are that

The discrimination objection is as follows. Some people who are physically, mentally, or emotionally impaired are so as the result of genetic factors they have inherited. Such impairment can result in disablement in our society. People with disabilities are often discriminated against by having fewer opportunities than other people. Be removing genetic disorders, and resulting impairment, it is true that gene therapy could contribute to removing one of the sources of discrimination and inequality in society. But the implicit assumption being made, the objection claims, is that people impaired through genetic factors need to be treated and made normal. The objection sees gene therapy as a form of discrimination against impaired people and persons with disabilities.

The irrelevance objection is that gene therapy on reproductive cells may in some cases already be superceded by in-vitro fertilization and selection of embryos. If a genetic disorder is such that can be detected in an early embryo, and not all embryos from the parent couple would have it, then have parents produce multiple embryos through in-vitro fertilization and implant only those free from the disorder. In such a case gene therapy would be unnecessary and irrelevant.

Following are some other important objections:

Gene therapy is becoming a reality as you read this. Genetic engineering for enhancement is still a ways off. Plenty of debate is sure to occur over both issues.

Read the rest here:
Gene Therapy and Genetic Engineering - University of Missouri