Category Archives: Gene Medicine

chairoij/ShutterStockImagine having your spleen removed, undergoing a double hip replacement, and receiving monthly blood transfusions to prevent severe pain attacks, all by the age of 13. That was the life of a teenager in France with sickle cell disease (SCD) until October 2014, when he received experimental gene therapy as part of a clinical study. Now, hes completely off all medications and his SCD is essentially gone, making him the hopeful poster child for the worlds first effective sickle cell disease therapy. (Dont these medical miracles that doctors cant explain.)

Standard treatments were not able to control his SCD symptoms [but] since receiving the stem cell transplant with LentiGlobin, he has been free from severe symptoms and has resumed normal activities, without the need for further transfusions, said study author Marina Cavazzana, MD, PhD, of Necker Hospital in Paris, France, where the trial was conducted, in a news release.

SCD is a inherited blood disorder where sufferers have sickle hemoglobin, an abnormal form of the oxygen-carrying protein which changes the shape of red blood cells (from a flexible disc shape to a rigid crescent one), making it hard for them to pass through blood vessels and often causing blockages that slow or stop the flow of oxygen-rich blood to nearby tissues, causing sudden and severe pain. Sickled red blood cells also die after 10 to 20 days, compared to normal ones which can live up to 120; this can cause the body to have trouble keeping up with red blood cell production, leading to anemia. A stem-cell transplant is currently the only curative option for patients, but fewer than 18 percent of patients are able to find a matching donor.

That is until now. The 13-year-old boy (known as Patient 1204) had bone marrow extracted, which was then genetically altered with the drug LentiGlobin BB305 so that his body made normal, healthy red blood cells instead of the sickle cells it was creating before. After just six months, the proportions of sickled red cells in his blood were significantly lower than those in untreated SCD patients. Now more than 15 months since the treatment, his body is still producing normal red blood cells and he hasnt experience any SCD-related episodes or hospitalizations, according to the study published in the New England Journal of Medicine.

Ive worked in gene therapy for a long time and we make small steps and know theres years more work. But here you have someone who has received gene therapy and has complete clinical remissionthats a huge step forward, Deborah Gill, PhD, of the gene medicine research group at the University of Oxford in England told BBC.

Scientists plan to test the drug on other sickle cell disease patients to see if the results are replicated.

MORE: This Grandmother Beat Cancer in a Groundbreaking 20-Minute Treatment

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Teen's Sickle Cell Disease Reversed - Reader's Digest - Reader's Digest

CHAMBERSBURG, Pa. - Virdis Medicine, a medical marijuana dispensary, has been working with the borough in Chambersburg in hopes to one day come to town.

Karen Diller is a loving mom who will do anything for her daughter 20 year-old daughter Karly.

Karly has CDKL5, a rare genetic disorder, which means her body is missing a gene and can not produce the protein that talks to her cells. As a result, Karly doesn't have the use of her hands or the ability to talk.

It started a whole bunch of testing and doctor visits and to no avail we could not find out what was wrong and there was no way to help her, said Diller, a Chambersburg resident.

Karly has had seizures ever since she was two weeks old. Diller says they tried different types of medicine and nothing worked until two-years-ago when they went to Langone University Hospital in New York City. Karly was given a CBD oil, a medical marijuana product.

It has lowered her seizures from multiple seizures a day to 60 to 70 percent fewer seizures, she now has days and consecutive days in a row without seizures, said Diller.

The trial in New York has been great, but the drive to New York from Chambersburg is five hours one way.

I don't know how long we're going to be able to continue the drive to New York City and how long she will actually be eligible for this trial, so Im very supportive of Virdis Medicine coming to Chambersburg, said Diller.

In addition to reducing seizures,CBD oil has made Karly calmer and better able to function. Diller says she will continue to advocate for Virdis Medicine.

I feel that could give so many people a better quality of the days that they have here, just like its helping her and I think we have to try, said Diller.

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A Chambersburg resident shares how Medical Marijuana has had a major impact on her daughter - WHAG

March 9, 2017 In the Department of Genomics at the Life & Brain research center: Dr. Andreas Forstner (seated at the front), associate professor (Privatdozent) Dr. Rupert Conrad and psychologist Stefanie Rambau. Credit: Katharina Wislsperger/UKB-Ukom

People with social anxiety avoid situations in which they are exposed to judgment by others. Those affected also lead a withdrawn life and maintain contact above all on the Internet. Around one in ten people is affected by this anxiety disorder over the course of their life. Researchers at the University of Bonn have now found evidence for a gene that is believed to be linked to the illness. It encodes a serotonin transporter in the brain. Interestingly, this messenger suppresses feelings of anxiety and depressiveness. The scientists want to investigate this cause more precisely and are thus looking for more study participants. The results will be published in the journal Psychiatric Genetics.

Heart palpitations, trembling and shortness of breath: those who suffer from social phobia avoid larger groups. Verbal tests or everyday arrangements are filled with fear - after all, other people could make a negative judgement. Those affected often avoid such situations for this reason. Contact is often easier over social media or anonymously over the Internet. Social phobias are among the psychiatric disorders that are triggered simultaneously by genetic and environmental factors. "There is still a great deal to be done in terms of researching the genetic causes of this illness," says Dr. Andreas Forstner from the Institute of Human Genetics at the University of Bonn. "Until now, only a few candidate genes have been known that could be linked to this."

Individual base pairs can vary in the DNA

Together with the Clinic and Policlinic for Psychosomatic Medicine and Psychotherapy at the University Hospital Bonn, Dr. Forstner is conducting a study into the genetic causes of social phobia. The research team investigated the DNA of a total of 321 patients and compared it with 804 control individuals. The focus of the scientists lay on what are known as single nucleotide polymorphisms (SNPs). "There are variable positions in the DNA that can exist to various degrees in different people," explains Dr. Forstner.

The cause of genetic illnesses often lies in the SNPs. It is estimated that more than thirteen million such changes exist in the human DNA. The scientists investigated a total of 24 SNPs that are suspected in the widest sense of being the cause of social phobias and other mental disorders. "This is the largest association study so far into social phobia," says associate professor (Privatdozent) Johannes Schumacher from the Institute of Human Genetics at the University of Bonn.

Patients provided information about their symptoms

Over the course of the study, scientists at the Clinic and Policlinic for Psychosomatic Medicine and Psychotherapy at the University Hospital Bonn will ask the patients about their symptoms and the severity of their social phobia. Their DNA is also examined using a blood sample. Whether there is a link between the signs of the illness and the genes is being investigated by the scientists using statistical methods. The evaluation of the previously collected data indicated that an SNP in the serotonin transporter gene SLC6A4 is involved in the development of social phobia.

This gene encodes a mechanism in the brain that is involved in transporting the important messenger serotonin. This substance suppresses, among other things, feelings of fear and depressive moods. "The result substantiates indications from previous studies that serotonin plays an important role in social phobia," says associate professor (Privatdozent) Dr. Rupert Conrad from the Clinic and Policlinic for Psychosomatic Medicine and Psychotherapy. Medications that block serotonin reuptake and increase the concentration of the messenger in the tissue fluid in the brain have already long been used to treat anxiety disorders and depression.

Subjects can participate in expanded study

The scientists now want to investigate more closely what the links are between the DNA and social phobia. "In order to achieve this goal, we need many more study participants who suffer from social anxiety," says the psychologist and study coordinator Stefanie Rambau from the Clinic and Policlinic for Psychosomatic Medicine and Psychotherapy at University Hospital Bonn. Information about the study is available at http://www.SocialPhobiaResearch.de. "Those who take part will help to research social phobia. This is the basis of better diagnosis and treatment procedures in the future," says Stefanie Rambau.

Explore further: Psychotherapy normalizes the brain in social phobia

More information: Andreas J. Forstner et al, Further evidence for genetic variation at the serotonin transporter gene SLC6A4 contributing toward anxiety, Psychiatric Genetics (2017). DOI: 10.1097/YPG.0000000000000171

Anxiety in social situations is not a rare problem: Around one in ten people are affected by social anxiety disorder during their lifetime. Social anxiety disorder is diagnosed if fears and anxiety in social situations significantly ...

Previous studies have led researchers to believe that individuals with social anxiety disorder/ social phobia have too low levels of the neurotransmitter serotonin. A new study carried out at Uppsala University, however, ...

Social phobia is the most common anxiety disorder of our time. But the current treatment regimen for patients with this diagnosis has not proven very effective. Norwegian and British researchers spent 10 years studying alternative ...

Anxiety disorders affect approximately one in six adult Americans, according to the National Institute of Mental Health. The most well-known of these include panic disorder, post-traumatic stress disorder, obsessive-compulsive ...

(Medical Xpress) -- A recent study from the Centre for Emotional Health, Macquarie University, has found children with social phobia are judged as less attractive and are less liked by their peers, than children without anxiety ...

Researchers often observe inadequate parenting, a negative emotional climate and household chaos in families of children with ADHD. A research group at Goethe University Frankfurt and the universities of Bremen, Heidelberg, ...

A study led by Ravi Bansal, PhD, and Bradley S. Peterson, MD, of The Saban Research Institute of Children's Hospital Los Angeles, has found structural differences in the cerebral cortex of patients with depression and that ...

Growing up with a pet can bring social, emotional and educational benefit to children and adolescents, according to a new University of Liverpool study. Youngsters with pets tend to have greater self-esteem, less loneliness, ...

Giving people time to think about cooperating on a task can have a positive effect if they are big-picture thinkers, but if they tend to focus on their own, immediate experience, the time to think may make them less cooperative, ...

There is no added benefit to using ketamine over a standard anaesthetic during electroconvulsive therapy (ECT) according to new research from the University of Aberdeen.

People who suffer from depression should participate in yoga and deep (coherent) breathing classes at least twice weekly plus practice at home to receive a significant reduction in their symptoms.

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Social phobia: Indication of a genetic cause - Medical Xpress - Medical Xpress

An international team of researchers has discovered that mutations in the human gene CWC27 result in a spectrum of clinical conditions that include retinal degeneration and problems with craniofacial and skeletal development. The results appear in the American Journal of Human Genetics.

CWC27 is a new disease-associated gene, said co-senior author Dr. Rui Chen, associate professor of molecular and human genetics at Baylor College of Medicine.

One of the goals of the Chen lab is to identify genes involved with human retinal disease, such as retinitis pigmentosa, a condition characterized by progressive development of night blindness and tunnel vision, sometimes from the early age of 2. Retinitis pigmentosa is the most common inherited disorder of the retina; it affects nearly 1 in 4,000 people, and more than 1 million are visually impaired around the world due to this untreatable disease.

In our search for genes linked to retinitis pigmentosa, we identified a patient with the condition more than two years ago, said co-first author Mingchu Xu, graduate student in molecular and human genetics in the Chen lab. We identified a frameshift mutation in CWC27. The patient did not have other conditions in addition to the vision problems. To study the condition, we mimicked the human mutation in a mouse model, and at 6 months of age the mice showed retinal degeneration and no other conditions, just as we had observed in the human patient.

CWC27 is one of more than 100 genes that participate in the formation and function of the spliceosome, a molecular machine that is involved in the correct expression of the proteins that carry out the functions of all the cells in the body. Until now, most disease-associated genes of the spliceosome had been involved in two non-overlapping conditions. For instance, mutations in certain proteins of the spliceosome cause syndromes that involve mainly craniofacial and skeletal conditions, while mutations in other spliceosome genes result only in retinitis pigmentosa. CWC27 seemed to belong to the second group of genes.

Surprising results

Interestingly, our collaborator Dr. Daniel Schorderet, director of the Institute for Research in Ophthalmology in Switzerland and co-senior author of the paper, was working with patients who have mutations in CWC27 and present with more severe clinical conditions than our patient, including craniofacial and skeletal problems in addition to problems with vision, Xu said.

When we looked at the clinical characteristics of all the patients, we did not anticipate that they would have mutations in the same gene. Only when we looked at the genes did we realize that the spectrum of clinical characteristic in the patients was the result of various mutations in the same gene, CWC27, Chen said.

By applying exome sequencing to multiple families and modeling the disease in two mouse models the researchers were able to appreciate the spectrum of clinical conditions that mutations in the same gene can cause.

This is the first time a mutation of a gene in the spliceosome has been described to result in an entire spectrum of clinical conditions, Xu said. To explain why our patient presented only with vision problems, we hypothesized that the mutation in our patients CWC27 was milder than those of other patients. By analyzing the results on mouse models and patient samples, we found that the mutant gene in our patient probably retains a residual function, while the genes in the patients of the other groups have a more severe loss of function.

This study also shows the power of collaboration within the genetics community when looking for new disease-associated genes, Xu said. Initially, we only identified one patient and then we collected more cases via two platforms, GeneMatcher and the European Retinal Disease Consortium. We would not have been able to present this interesting story without the contributions of researchers from nine countries. With exome sequencing accessible to more patients and researchers, these platforms will most likely speed up the process of finding the genetic causes of human diseases.

Seea complete list of authors and their affiliations and the financial support for this project.

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Mutations in CWC27 result in spectrum of conditions - Baylor College of Medicine News (press release)

Science Daily

Study parses influence of genes, environment in metabolic disease Science Daily By comparing two strains of mice -- one that becomes obese and diabetic on a high-fat diet and another resistant to a high-fat regimen -- researchers from the Perelman School of Medicine at the University of Pennsylvania identified genome-wide changes ...

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Study parses influence of genes, environment in metabolic disease - Science Daily

March 8, 2017

Today, in vitro fertilization provides a way for couples to avoid passing potentially disease-causing genes to their offspring. A couple will undergo genetic screening. Tests will determine whether their unborn children are at risk. If embryos created through IVF show signs of such a genetic mutation, they can be discarded.

Flash forward a few years, and, instead of being discarded, those embryos can be repaired with new gene editing technologies. And those repairs will affect not only those children, but all their descendants

This is definitely new territory, said Pasquale Patrizio, M.D., director of the Yale Fertility Center and Fertility Preservation Program. We are at the verge of a huge revolution in the way disease is treated.

We are at the verge of a huge revolution in the way disease is treated.

In a move that seems likely to help clear the path for the use of gene editing in the clinical setting, on February 14 the Committee on Human Gene Editing, formed by the National Academy of Medicine and the National Academy of Sciences, recommended that research into human gene editing should go forward under strict ethical and safety guidelines. Among their concerns were ensuring that the technology be used to treat only serious diseases for which there is no other remedy, that there be broad oversight, and that there be equal access to the treatment. These guidelines provide a framework for discussion of technology that has been described as an ethical minefield and for which there is no government support in the United States.

A main impetus for the committees work appears to be the discovery and widespread use of CRISPR-Cas9, a defense that bacteria use against viral infection. Scientists including former Yale faculty member Jennifer Doudna, Ph.D., now at the University of California, Berkeley, and Emmanuelle Charpentier, Ph.D., of the Max Planck Institute for Infection Biology in Berlin, discerned that the CRISPR enzyme could be harnessed to make precision cuts and repairs to genes. Faster, easier, and cheaper than previous gene editing technologies, CRISPR was declared the breakthrough of the year in 2015 by Science magazine, and has become a basic and ubiquitous laboratory research tool. The committees guidelines, said scientists, physicians, and ethicists at Yale, could pave the way for thoughtful and safe use of this and other human gene editing technologies. In addition to CRISPR, the committee described three commonly used gene editing techniques; zinc finger nucleases, meganucleases, and transcription activator-like effector nucleases.

Patrizio, professor of obstetrics, gynecology, and reproductive sciences, said the guidelines are on the mark, especially because they call for editing only in circumstances where the diseases or disabilities are serious and where there are not alternative treatments. He and others cited such diseases as cystic fibrosis, sickle cell anemia, and thalassemia as targets for gene editing. Because they are caused by mutations in a single gene, repairing that one gene could prevent disease.

Peter Glazer, M.D. 87, Ph.D. 87, HS 91, FW 91, chair and the Robert E. Hunter Professor of Therapeutic Radiology and professor of genetics, said, The field will benefit from guidelines that are thoughtfully developed. This was a step in the right direction.

The panel recommended that gene editing techniques should be limited to deal with genes proven to cause or predispose to specific diseases. It should be used to convert mutated genes to versions that are already prevalent in the population. The panel also called for stringent oversight of the process and for a prohibition against use of the technology for enhancements, rather than to treat disease. As physicians, we understand what serious diseases are. Many of them are very well known and well characterized on a genetic level, Glazer said. The slippery slope is where people start thinking about modifications in situations where people dont have a serious disorder or disease.

Mark Mercurio, M.D., professor of pediatrics (neonatology), and director of the Program for Biomedical Ethics, echoed that concern. While he concurs with the panels recommendations, he urged a clear definition of disease prevention and treatment. At some point we are not treating, but enhancing. This in turn, he said, conjures up the nations own medical ethical history, which includes eugenics policies in the early 20th century that were later adopted in Nazi Germany. This has the potential to help a great many people, and is a great advance. But we need to be cognizant of the history of eugenics in the United States and elsewhere, and need to be very thoughtful in how we use this technology going forward, he said.

The new technology, he said, can lead to uncharted ethical waters. Pediatric ethics are more difficult, Mercurio said. It is one thing to decide for yourselfis this a risk Im willing to takeand another thing to decide for a child. It is another thing still further, which we have never had to consider, to decide for future generations.

Myron Genel, M.D., emeritus professor of pediatrics and senior research scientist, served on Connecticuts stem cell commission and four years on the Health and Human Services Secretary's Advisory Committee on Human Research Protections. He believes that Connecticuts guidelines on stem cell research provide a framework for addressing the issues associated with human gene editing. There is a whole regulatory process that has been evolved governing the therapeutic use of stem cells, he said. There are mechanisms that have been put in place for effective local oversight and national oversight for stem cell research.

Although CRISPR has been the subject of a bitter patent dispute between Doudna and Charpentier and The Broad Institute in Cambridge, Mass., a recent decision by the U.S. Patent Trial and Appeal Board in favor of Broad is unlikely to affect research at Yale and other institutions. Although Broad, an institute of Harvard and the Massachusetts Institute of Technology, can now claim the patent, universities do not typically enforce patent rights against other universities over research uses.

At Yale, scientists and physicians noted that gene editing is years away from human trials, and that risks remain. The issue now, said Glazer, is How do we do it safely? It is never going to be risk-free. Many medical therapies have side effects and we balance the risks and benefits. Despite its effectiveness, CRISPR is also known for whats called off-target risk, imprecise cutting and splicing of genes that could lead to unforeseen side effects that persist in future generations. CRISPR is extremely potent in editing the gene it is targeting, Glazer said. But it is still somewhat promiscuous and will cut other places. It could damage a gene you dont want damaged.

Glazer has been working with a gene editing technology called triple helix that hijacks DNAs own repair mechanisms to fix gene mutations. Triple helix, as its name suggests, adds a third strand to the double helix of DNA. That third layer, a peptide nucleic acid, binds to DNA and provokes a natural repair process that copies a strand of DNA into a target gene. Unlike CRISPR and other editing techniques, it does not use nucleases that cut DNA. This just recruits a process that is natural. Then you give the cell this piece of DNA, this template that has a new sequence, Glazer said, adding that triple helix is more precise than CRISPR and leads to fewer off-target effects, but is a more complex technology that requires advanced synthetic chemistry.

Along with several scientists across Yale, Glazer is studying triple helix as a potential treatment for cystic fibrosis, HIV/AIDS, spherocytosis, and thalassemia.

Adele Ricciardi, a student in her sixth year of the M.D./Ph.D. program, is working with Glazer and other faculty on use of triple helix to make DNA repairs in utero. She also supports the panels decision, but believes that more public discussion is needed to allay fears of misuse of the technology. In a recent presentation to her lab mates, she noted that surveys show widespread public concern about such biomedical advances. One study found that most of those surveyed felt it should be illegal to change the genes of unborn babies, even to prevent disease.

There is, I believe, a misconception of what we are using gene editing for, Ricciardi said. We are using it to edit disease-causing mutations, not to improve the intelligence of our species or get favorable characteristics in babies. We can improve quality of life in kids with severe genetic disorders.

This article was submitted by John Dent Curtis on March 8, 2017.

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Gene editing opens the door to a revolution in treating and preventing disease - Yale News

Genetic changes in the cells lining the inside of the nose might someday help doctors diagnose lung cancer, a recent study suggests.

"The idea that you don't have to sample the disease tissue but can diagnose presence of disease using relatively accessible cells that are far from the tumor . . . is a paradigm that can impact many cancers," Dr. Avrum Spira from Boston University School of Medicine, a member of the study team, told Reuters Health by email.

The layer of cells that covers the surfaces of the body and lines the cavities is known as the epithelium. Researchers found that distinctive changes in gene activity in the nasal epithelium of lung cancer patients closely parallel the changes seen in lung epithelium and can distinguish between benign lung disease and cancer.

"I think the most interesting finding was the genomic changes in the nasal epithelium of lung cancer patients mirror so closely those found in the lower airway," Spira said.

The researchers thought the nose would be "a reasonable surrogate for the 'field of injury' in the bronchial airway," he added, but the surprisingly strong concordance between the nose and lower airway gave them the encouragement to develop a nasal biomarker for lung cancer detection.

"Pulmonary nodules represent a growing diagnostic dilemma in the U.S. as we have started to screen for lung cancer," Spira said. "A nasal swab that is highly sensitive for lung cancer in this setting would enable physicians to avoid unnecessary invasive biopsies in nodule patients who are unlikely to have lung cancer."

Past research has found that gene expression profiles from normal bronchial epithelial cells can distinguish smokers and former smokers with lung cancer from individuals with benign lung disease, and that nasal and bronchial epithelium respond similarly to tobacco smoke.

Spira's team sought to determine whether cancer-associated gene expression in the nasal epithelium might be useful for detecting lung cancer in current and former smokers.

They identified 535 genes that had different activity patterns in the nasal epithelium of patients with lung cancer versus those with benign disease.

Cancer-associated gene changes correlated significantly between nasal epithelium and bronchial epithelium samples, and the genes that were more active in nasal epithelium of patients with lung cancer were among the genes whose activity was most increased in bronchial epithelium of patients with cancer.

When researchers compared models doctors might use to determine the likelihood of lung cancer, nasal gene activity was more accurate than clinical risk factors alone for diagnosing lung cancer, according to the Journal of the National Cancer Institute report.

The combination of clinical factors and gene activity score accurately predicted cancer 91 percent of the time, compared to 79 percent for the model based on risk factors. The combined model also had 85 percent accuracy differentiating lung cancer from benign disease, compared to 73 percent.

"One of the big-picture messages for physicians is that molecular tests like this one are emerging as part of precision medicine approaches for early cancer detection," Spira said.

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Gene activity in the nose may signal lung cancer - Fox News

TUESDAY, March 7, 2017 (HealthDay News) -- A drug used to treat Alzheimer's disease should not be prescribed to people with milder mental impairment without first giving them a genetic test, a new study urges.

The drug is donepezil (brand name: Aricept).

Donepezil could speed mental decline in someone with mild cognitive impairment who has a specific genetic variation, according to Sophie Sokolow, an associate professor at the UCLA School of Nursing.

She and her colleagues found that patients with the K-variant of the butyrylcholinesterase (BChE) gene who took donepezil deteriorated faster than those who took a placebo.

Donepezil is approved in the United States to treat Alzheimer's disease but not mild cognitive impairment -- the stage between normal age-related decline and dementia. However, doctors often prescribe it "off-label" for patients with mild cognitive impairment, the study authors said.

For this study, the researchers examined data from a U.S. government-funded study published in 2005 that assessed donepezil as a possible treatment for mild cognitive impairment.

The findings reinforce the importance of physicians discussing the possible benefits and risks of donepezil with their patients, the researchers said in a university news release.

The study was published recently in the Journal of Alzheimer's Disease. Funding was provide by the U.S. National Institute on Aging.

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Study: Gene Test Needed Before Using Alzheimer's Drug 'Off-Label' - Journal Gazette and Times-Courier

NEW YORK (GenomeWeb) Before the National Institutes of Health can begin to genetically test participants within its precision medicine initiative, it will have to figure out what results to return, how to minimize reporting false positives, and how to provide counseling to help them navigate the often uncertain and evolving evidence on genetic information.

And the project will have to figure out how to do all this on an unprecedented scale, for a million participants that the All of Us Research Program hopes to enroll over the next four years.

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Precision Medicine Project Mulls How to Return Genetic Test Results to 1 Million Participants - GenomeWeb

Broad Institute wins gene-editing patent case, boosting Editas shares Intellia moves into new HQ, says it plans to double headcount

John Leonard, executive vice president of R&D at Intellia

Cambridge-based Intellia Therapeutics had the biggest IPO of any local biotech in 2016, but shares of the company have lagged in recent months, and it has often been overshadowed by its gene-editing rival in Kendall Square, Editas Medicine.

On Wednesday, though, Intellia (Nasdaq: NTLA) wrested back the spotlight, announcing promising preclinical data from a study of its technology in mice that offers a pathway to potential trials involving humans.

John Leonard, executive vice president of R&D at Intellia

The company and partner Regeneron Pharmaceuticals (Nasdaq: REGN) have been developing a potential treatment for a rare hereditary disease called Transthyretin Amyloidosis, or ATTR. Intellias approach involves editing the defective genes that cause the buildup of the mutant protein responsible for the disease. The gene-editing tool, dubbed CRISPR/Cas9, is injected via lipid nanoparticles.

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Intellia R&D head says new gene-editing data shows path to human trials - Boston Business Journal