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News Release
NHGRI is awarding Genomic Innovator Awards to nine institutions to support the research of 12 early career scientists in the field of genomics. The awards will total up to $27 million over five years.
… Stanford, California Apply CRISPR tools to understand gene regulation related to common, complex diseases.  Audrey … medicine, CRISPR, non-coding DNA and RNA sequences, gene regulation, single-cell biology, chromatin state …
Fact Sheet
Learn about the different types of hemoglobin.
… To understand sickle cell disease and gene therapy , you should first know about blood cells and the … before birth (during fetal development). After birth, a gene that codes for a blocking protein (BCL11A) is turned on. …
Event
A joint NHGRI-NCATS hosted meeting bringing together researchers, clinicians, families and patient advocates from around the world to discuss the natural history, disease pathophysiology, genetics and treatments for rare inborn metabolic errors.
… A. Chapman, M.D., Ph.D. 3:10 - 3:30 p.m. Systemic AAV gene therapy for propionic acidemia (PA) Randy Chandler, Ph.D. … 2:30 p.m. Treatment of CBS Deficient Mice by AAV-Mediated Gene Therapy Warren Kruger, Ph.D. 2:30 - 3:00 p.m. Break  …
News Release
NHGRI has selected Charles Venditti, M.D., Ph.D., as chief of its Metabolic Medicine Branch within the Institute’s Intramural Research Program. In this role, he will provide leadership and guidance on research into genetic diseases that disrupt metabolism.
… treatments for these metabolic disorders, including gene therapies and pharmaceuticals. Branch investigators also …
The Genomics Landscape
In the January 2024 issue of The Genomics Landscape, NHGRI Director Eric Green announces the appointment of Dr. Adam Phillippy as the founding director of the Center for Genomics and Data Science Research (CGDSR).
… Food and Drug Administration (FDA), which approved two gene therapies for the treatment of sickle cell disease. … region of the angiotensin-converting enzyme 2 (ACE2) gene, which encodes the cellular receptor for SARS-CoV-2. In … Data Science Research NIH statement on new FDA-approved gene therapies for sickle cell disease NHGRI searches for new …
The Genomics Landscape
In the January 2018 edition of The Genomics Landscape, NHGRI Director Eric Green features the NIH Intramural Sequencing Center.
… runners-up include three relevant to genomics: " pinpoint gene editing ," " a cancer drug's broad swipe ," and " gene therapy triumph ." Nature's  list of Science Events that …
Profile
Carlos Ferreira, M.D., staff clinician in NHGRI's Metabolic Medicine Branch, knows the catalogue of rare skeletal disorders well. With hundreds of known rare bone disorders and more to be discovered, Dr. Ferreira breaks down the challenges of skeletal genomics.
… and approaches currently being developed, such as gene therapy and the use of small molecules. By improving our …
The Genomics Landscape
In the July 2024 issue of The Genomics Landscape, NHGRI Director Eric Green welcomes the inaugural class of the ASHG-NHGRI Genomics and Public Service Fellowship Program.
… best,  … Effect of genomic and cellular environments on gene expression noise Hong, et al. Genome Biol. , 2024 May, … proteins. One of the first steps of this process, called gene expression, involves copying DNA into RNA. While this is … for targeting genome editing, such as that used for gene therapy.  This research was funded in part by the NIH with a …
The Genomics Landscape
In the April 2019 edition of The Genomics Landscape, NHGRI Director Eric Green previews the redesigned genome.gov website and its new features.
… Lung, and Blood Institute, who is working to develop a gene therapy-based treatment for sickle cell disease. This … Alcohol Use and Adult Anxiety  Largest-Ever Alzheimer's Gene Study Brings New Answers  Mood-Altering Messenger Goes …
News Release
Top genome scientists from over 20 countries have joined forces in an effort to improve cooperation and coordination of genomic medicine research worldwide.
… widespread genetic testing against a potentially fatal, gene-linked skin disease that occurs with unusually high …