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Research at NHGRI
The Democratizing Education for Sickle Cell Disease Gene Therapy project developed patient-focused educational materials for the sickle cell community.
… In December 2023, the U.S. Food and Drug Administration (FDA) approved two gene therapies to treat sickle cell …
Fact Sheet
Sickle cell disease resources for patients
… What is Gene Therapy?  U.S. Food and Drug Administration (FDA)  Gene Therapies for Sickle Cell Disease   Cure Sickle … suspension  DailyMed, National Library of Medicine   FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease   Casgevy  FDA Lovotibeglogene autotemcel  Lovotibeglogene autotemcel …
Staff
Dr. Michael Erdos is an Associate Investigator in the Molecular Genetics Section of the Center for Precision Health Research.
… treatment strategies are in the application process for FDA approval. In the context of complex genetic disease, Dr. … stiffness, and ultimately a modest extension in lifespan. FDA approved lonafarnib (a farnesyltransferase inhibitor) for … Chromatin stretch enhancer states drive cell-specific gene regulation and harbor human disease risk variants . Proc. …
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.
… and overall policies on such issues as data-sharing, regulation and cost-benefit analysis. Not coincidentally, … Institutes of Health and the Food and Drug Administration (FDA) sponsored a workshop in March on this disease. Videos of …
Policy Issues
There are laws and policies that serve to protect the privacy of individuals' genomic information.
… their genetic information. DTC genetic tests have limited regulation , and the growth of the DTC genetic testing …
National Advisory Council for Human Genome Research
The Director's Report-Related Documents contain links to materials supplemental to Dr. Eric Green's Director's Report presentation to the National Advisory Council for Human Genome Research in February 2024.
… 10 Re-Envisioning NIH-Supported Postdoctoral Training 11 FDA Proposed Rule for LDT Oversight and Regulation FDA Proposed Rule Association for Molecular Pathology …
The Genomics Landscape
In the September 2023 edition of The Genomics Landscape, NHGRI Director Eric Green, recaps the April 2023 virtual reunion of the leaders of the five genome-sequencing centers (G5), where they tell the untold story on how they ushered the Human Genome Project across the finish line in 2003.
… trial not allowed and clinical trial required NIH, CDC and FDA for small business innovation research grant applications …
Fact Sheets
Cloning describes a number of different processes that can be used to produce genetically identical copies of a biological entity.
… experts in cloning, the U.S. Food and Drug Administration (FDA) decided in January 2008 that meat and milk from cloned … goats, are as safe as those from non-cloned animals. The FDA action means that researchers are now free to using …
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.
… Rob Stein GDPR: Crossing the Data Sharing Bridge, One Regulation at a Time Genome: Unlocking Life's Code March 2019 …
Clinical Research
The Institutional Review Board (IRB) is responsible for protecting the rights and welfare of the human subjects of research protocols that are conducted by intramural investigators in NHGRI and a number of other institutes.
… (OHRP) OHRP Approved Federal-Wide Assurances (FWAs) FDA Guidance for IRBs, Clinical Investigators and Sponsors   FDA Investigator-Initiated Investigational New Drug (IND) …