Learn more about the impact on rare disease diagnosis for conditions with a genetic component, and the potential to target a disease at its basis. Dr. P.J. Brooks, Ph.D., Deputy Director, National Institute of Health and Dr. Anne R. Pariser, M.D., Director, Office of Rare Disease Research, National Center for Advancing Translational Sciences discuss our change in understanding of rare diseases and the science to treat these diseases - developing targeted and personalized therapies are exciting.

This video is part of a Global Genes program, Platforms of Hope, detailing advances in gene therapy and gene editing. For more resources, visit [ Ссылка ].

свернуть