Homology Medicines, Inc.
ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR IDS GENE TRANSFER AND METHODS OF USE THEREOF

Abstract:

Provided are adeno-associated virus (AAV) compositions that can restore IDS gene function in cells, and methods for using the these AAV compositions to treat disorders associated with reduction of IDS gene function (e.g., Hunter syndrome). Also provided are compositions, systems and methods for making the AAV compositions.