SELF-COMPLEMENTARY AAV-MEDIATED DELIVERY OF INTERFERING RNA MOLECULES TO TREAT OR PREVENT OCULAR DISORDERS

摘要

The invention provides methods for delivering interfering RNA molecules to an eye of a patient to treat ocular disorders. In particular, the methods of the invention comprise the use of a self-complementary adeno-associated (scAAV) viral vector that can deliver an interfering RNA molecule to an eye of a patient to inhibit expression of a gene that is associated with an ocular disorder.