Search Results
Policy Issues
Most ethical discussions about genome editing center on human germline editing because changes are passed down to future generations.
Fact Sheets
Genetics refers to the study of genes and their roles in inheritance. Genomics refers to the study of all of a person's genes (the genome).
Staff
Cynthia Tifft, M.D., Ph.D. is the deputy clinical director for the National Human Genome Research Institute.
… will be important for ongoing pre-clinical testing of therapeutics for GM1 patients. GM1 gangliosidosis is a rare …
News Release
Funding to advance understanding of RNA biology and development of RNA-based technologies
… advances have harnessed RNA to develop technologies and therapeutics such as small interfering RNA-based drugs and …
Staff
Dr. Ellen Sidransky is a pediatrician and clinical geneticist in the Medical Genetics Branch at the National Human Genome Research Institute.
… cell model provides a tool to probe pathophysiology and therapeutics for Gaucher disease. Disease Models & …
Media Availability
Researchers have created a promising new method to accurately and more comprehensively analyze and interpret DNA sequence information from cancer patients.
Staff
Dr. Joshua Denny is the head of the NHGRI Precision Health Informatics Section and Chief Executive Officer of the National Institutes of Health’s All of Us Research Program.
Policy Issues
Genome editing is currently being applied to research on cancer, mental health, rare diseases, and many other disease areas.
For Patients and Families
A rare disease is generally considered to be a disease that affects fewer than 200,000 people in the United States at any given time.
… Institutes of Health (NIH) launched an effort, called the Therapeutics for Rare and Neglected Diseases program , to …
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.