Search Results
Policy Issues
Genome editing is a method that lets scientists change the DNA of many organisms, including plants, bacteria, and animals.
… are two different categories of gene therapies: germline therapy and somatic therapy. Germline therapies change DNA in reproductive cells … these cells affect only the person who receives the gene therapy. In 2015, scientists successfully used  somatic gene …
Policy Issues
Scientists have edited genomes for many years, but CRISPR technology has improved the speed, cost, accuracy, and efficiency of genome editing
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… aid the development of new treatments, including gene therapy . … Scientists have had the knowledge and ability to … Genome Editing, Genomic Technology, Gene Editing, Gene Therapy, DNA Editing … Scientists have edited genomes for …
Policy Issues
Patients with genetic disorders and members of the public have diverse about germline genome editing.
… of 2016, passed in December 2015, prohibits the Food and Drug Administration (FDA) from reviewing research that … … Germline Genome Editing, Gene Editing, DNA Editing, Gene Therapy, Genetic Disorder, Genetic Disease, CRISPR-Cas9, …
For Patients and Families
The Genetic and Rare Diseases Information Center helps people find useful information about genetic and rare diseases.
… and rare diseases ​ … Genetic Disease, Rare Disease, Gene Therapy, Diagnosing A Disease, DNA Sequence, Genome Sequence, …
15 Ways Genomics Influences Our World
Advances in genomics are helping you access information about your genome from your home.
… you are interested in been approved by  the U.S. Food and Drug Administration (FDA) if it is about your health? How … Limitations To DTC Genomic Testing, Regulations, Food And Drug Administration (FDA) … Advances in genomics are helping …
Fact Sheet
Your mental and physical health are deeply connected. Mental health is important for your health and quality of life. 
…  There are many unknowns about what to expect from a gene therapy clinical trial. Your thoughts, feelings, beliefs and … for your mental and physical health as you consider a gene therapy clinical trial.  Focus on your thoughts and … of participation Check out the sickle cell disease gene therapy FAQ Cost What costs does the clinical trial cover? …
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. … David S. Rosenblatt, M.D. 10:30 - 10:45 a.m. High-Dose B12 Therapy for cblC Deficiency Irini Manoli, M.D., Ph.D. 10:45 - … p.m. Treatment of CBS Deficient Mice by AAV-Mediated Gene Therapy Warren Kruger, Ph.D. 2:30 - 3:00 p.m. Break  Session …
The Genomics Landscape
In the January 2018 edition of The Genomics Landscape, NHGRI Director Eric Green features the NIH Intramural Sequencing Center.
… to genomics: " pinpoint gene editing ," " a cancer drug's broad swipe ," and " gene therapy triumph ." Nature's  list of Science Events that … Research Dr. Collins Discusses Advances in Gene Therapy with the Associated Press Unlocking Life's Code : …
Event
On February 16, 2023, the National Human Genome Research Institute (NHGRI) held a follow-up working group consultation after the 14th Genomic Medicine meeting, Genomic Medicine XIV: Genomic Learning Healthcare Systems.
… It almost never gets used. We have CDS for >30 gene-drug pairs in our EHR; this may be relevant, but is not … a consultservice. We have a physician who is expert in drug interactions and PGx, Tom Callaghan, M.D., Ph.D., who has a special clinic setup for difficult cases of drug therapy adverse events and consults for PGx. In our Oncology …
Research Training
A fellowship that will prepare fellows to use genetic, genomic and pharmacogenomic approaches to advance minority health and health equity.
… Genome Research Institute (NIH NHGRI) and the Food and Drug Administration’s Office of Minority Health and Health … working closely with mentors at both FDA OMHHE, Center for Drug Evaluation and Research (CDER) and NIH NHGRI, providing …