ADENO-ASSOCIATED VIRUS-MEDIATED CRISPR-Cas9 TREATMENT OF OCULAR DISEASE

摘要

Disclosed herein are compositions and methods of treating and/or correcting ocular disease in a subject, such as a mammal (e.g., human) eye using an Adeno- associated virus (AAV) system. The AAV system employs a nucleic acid encoding a CRISPR-Cas9 system for targeted gene disruption or correction.