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News Release
NIH researchers compared a new genetic animal model of Down syndrome to the standard model and found the updated version to be enhanced.
… France, removed these extra 45 genes using CRISPR gene-editing technology. Dr. Bianchi’s group then compared the two …
News Release
Using a recently developed DNA base-editing technique, researchers correct accelerating aging disorder.
… Ganguly, Ph.D. … Using a recently developed DNA base-editing technique, researchers correct accelerating aging … Progeria is caused by a mutation in the nuclear lamin A gene in which one DNA base C is changed to a T. Researchers … is caused by a mutation in the nuclear lamin A (LMNA ) gene in which one DNA base C is changed to a T. This change …
News Release
Researchers and clinicians are sequencing human genomes faster than ever, so considering the societal implications of genomic data is even crucial.
… many users were interested in learning more about how gene editing technologies, like CRISPR-Cas9, could enhance the … minds given all the attention and promise of CRISPR gene editing technology. It's important to keep in mind that …
Research at NHGRI
The Metabolic Medicine Branch houses clinical and laboratory research programs that focus on the development of new therapies for patients with genetic disorders.
… Our investigators and their research teams develop gene addition therapy and gene editing approaches to treat metabolic disorders, study immune …
News Release
The NHGRI Short Course in Genomics brings the latest genomics science to the classroom.
… topics, including genome sequencing, CRISPR and other gene-editing methods, the human microbiome, and ethical and …
Staff
Dr. Randy Chandler is an associate investigator in the Organic Acid Research Section of the Metabolic Medicine Branch in NHGRI.
… research focused on adeno-associated virus (AAV) gene therapy for the treatment of hemophilia A, a genetically … Dr. Chandler’s research has focused on the efficacy of gene therapy as a treatment for rare genetic disorders, such … therapy and the other, AAV-mediated nuclease-free genome editing. In addition, he is a member of NCATS’s Platform …
The Genomics Landscape
In the December 2018 edition of The Genomics Landscape, NHGRI Director Eric Green recaps progress from NHGRI strategic planning process events in 2018.
… Research NIH Researcher Presents Encouraging Results for Gene Therapy for Severe Sickle Cell Disease Blast Off! … Genomes for Precision Medicine Statement on Claim of First Gene-Edited Babies by Chinese Researcher NHGRI's Dr. Andy …
News Release
DNA Day Reddit “Ask Me Anything” prompts rousing discussion.
… or multiple generations. We have two copies of every gene (except for those found only on the Y chromosome), and … that a person can have one defective copy of the related gene and one normal copy of the gene and won’t show any … not least, why should we even do it? The CRISPR/Cas9 gene editing system allows scientists to modify DNA at specific …
News Release
ENCODE program directors in the Division of Genome Sciences turned to Reddit to answer questions from the Reddit community.
… these sequences are so conserved. - Nadav CRISPR-Cas9, a gene editing tool which can be programmed to target specific … immortalized cell lines. However, it is still hard to do gene editing directly in a physiologically relevant way in …
Fact Sheet
Sickle cell disease resources for patients
… welcomes your feedback about the sickle cell disease gene therapy resources. Please email your comments or … They are meant to promote your general understanding of gene therapy for sickle cell disease. We encourage you to use … the Difference Between Gene Therapy, Cell Therapy and Gene Editing? (video) American Society of Gene & Cell Therapy …