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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 …
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).
Policy Issues
Most ethical discussions about genome editing center on human germline editing because changes are passed down to future generations.
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.
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 …
For Health Professionals
Archives of ISCC-PEG webinars, articles, case studies and more.
… and allopurinol Type of Case Study: Genomic-based therapeutics, Pharmacogenomic In collaboration with the …
Staff
Dr. Amanda Ombrello is a clinical researcher in the Inflammatory Disease Section of NHGRI's Medical Genetics Branch.