CLINICAL DEVELOPMENT OF AAV VECTORS

Abstract

Impressive progress has been made in clinical development of adeno-associated virus (AAV) vectors over the last 15 years in more than 40 clinical trials, involving many hundreds of subjects with vector delivery by many different routes and at a wide range of doses. Preclinical expectations, including an excellent safety profile, the tendency to remain at a local delivery site, and the persistence of expression for years after delivery, are being borne out in clinical trials. Immune responses to the vectors in human subjects, less well anticipated by preclinical studies, are now being examined in current AAV vector clinical trials, in order to determine in what circumstances such responses might occur or be problematic. Development of AAV vectors is likely to continue to be an expanding field. The current successes in the subjects with retinal degeneration and Parkinson's disease are highly encouraging for AAV vectors and for the whole field of gene therapy.