PTC Therapeutics, Inc.
VECTOR AND METHOD FOR TREATING ANGELMAN SYNDROME

Abstract:

One aspect described herein relates to a recombinant adeno-associated virus (rAAV) vector and a method for use thereof or treating Angelman Syndrome. Another aspect described herein is a UBE3A rAAV vector and method for use thereof for treating a UBE3A deficiency, e.g. Angelman syndrome, in humans.