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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 …
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 …
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 …
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 …
Event
We invite scholars to submit ideas to be one of six to seven speakers at our symposium, “Irreducible Subjects: Disability and Genomics in the Past, Present, and Future”, with an additional opportunity to join a collected set of papers on the same theme.
… and enhancement, as well as how some rhetoric around gene editing reflects the same utopian valorization of the … medicine, advancements in reproductive technologies and gene therapies have had enormous positive benefits for … racism, eugenics, and ableism Reproductive technologies Gene editing and enhancement Stigma and contemporary …
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 … Gene Therapy and Sickle Cell Disease (video) American …
The Genomics Landscape
In the January 2020 edition of The Genomics Landscape, NHGRI Director Eric Green highlights the newly developed polygenic risk scores webpage, which provides graphical and textual information about polygenic risk scores, describes polygenic traits, introduces how polygenic risk scores can be calculated, and explains how such scores can be interpreted.
… (those that are caused by variants in more than one gene), such as coronary heart disease, diabetes, and … shares NIH’s perspective on the potential of CRISPR genome-editing systems with Southwestern Medical Foundation’s … of the All of Us Research Program NIH Director on human gene editing: 'We must never allow our technology to eclipse …
The Genomics Landscape
In the December 2019 edition of The Genomics Landscape, NHGRI Director Eric Green highlights Ben Solomon joining NHGRI as Clinical Director.
… Genomics Research Gene-editing advance puts more gene-based cures within reach Notable accomplishments in …
News Release
Researchers from the NHGRI-funded Impact of Genomic Variation on Function Consortium are exploring how genomic variation affects genome function.
… test variants to determine which ones can affect gene expression in common human diseases. Hyejung Won, Ph.D. … variants in the regulatory elements increase or decrease gene activity and how the variants are affecting traits in … first. Then, they will use technologies based on the gene editing system CRISPR to add or change variants in the …
Fact Sheet
With increasing complexity in genomic data, researchers are turning to artificial intelligence and machine learning as ways to identify meaningful patterns for healthcare and research purposes.
… learning. Using deep learning to improve the function of gene editing tools such as CRISPR . These are just a few ways by …