Application Area: Liver Disease
10 Results for 'Liver Disease'
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...
Clinical studies take years to complete and testing a single compound can cost more than $2 billion. Meanwhile, innumerable animal lives are lost, and the process often fails to predict human responses because traditional animal models often do not accurately mimic human pathophysiology. For these reasons, there is a broad need for alternative ways to...