Homology Medicines, Inc.
Adeno-Associated Virus Compositions for ARSA Gene Transfer and Methods of Use Thereof

Abstract:

Provided herein are adeno-associated virus (AAV) compositions that can express an arylsulfatase A (ARSA) polypeptide in a cell, thereby restoring the ARSA gene function. Also provided are methods of using the AAV compositions, and packaging systems for making the AAV compositions.