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News Release
The U.S. National Institutes of Health (NIH) has long invested in basic genetics and genomics research, clinical trials, as well as translational medicine and social science studies, to advance our understanding of this widespread illness to help develop effective therapies.
… the U.S. Food and Drug Administration (FDA) approved two gene therapies for the treatment of sickle cell disease in … One of the new sickle cell treatments uses the CRISPR gene-editing system, a first for humans in the U.S. NIH …
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 …