9 Results for 'Skin'
AAV-Engineering For Advanced Gene Therapy
Gene therapy with AAV (adeno-associated virus) as a delivery vehicle is a leading therapeutic modality for genetic disorders that is approved in Europe for the metabolic disease Familial Lipoprotein Lipase Deficiency. It is currently being tested in clinical trials in the U.S. to treat other rare conditions like the retinal Leber Congenital Amaurosis and the...
Video/AnimationFluorescent in situ SequencingIn this video, George Church, Ph.D., a Core Faculty member at the Wyss Institute and Professor of Genetics at Harvard Medical School, explains how fluorescent in situ sequencing could lead to new diagnostics that spot the earliest signs of disease, and how it could help reveal how neurons in the brain connect and function. Credit:...