University of Pennsylvania

University of Southern California

Mcgill University

Devin McDougald

Jean Bennett

Junwei Sun

Ji Yun Song

Ahmara Gibbons Ross

Nancy Braverman

Catherine Argyriou

Joseph Hacia

Kenneth Shindler

Compositions and methods are provided for treating ocular neuropathy in a subject. In one aspect, a recombinant adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding NRF2. In another aspect, a recombinant adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding SIRT1. In desired embodiments, the subject is human, cat, dog, sheep, or non-human primate.

Gene therapy for ocular disorders

Methods and compositions are provided for treatment of peroxisomal biogenesis disorders (PBDs). More particularly, recombinant adeno-associated viruses (rAAV) provided in the form of compositions are used to deliver a nucleic acid encoding human PEX1 to host cells. The rAAVs comprise a AAV capsid, and packaged therein a vector genome comprising an AAV 5' inverted terminal repeat (ITR) sequence; a promoter; a coding sequence encoding a human PEX1; and an AAV 3&#x2032; ITR.

Gene therapy for treating peroxisomal disorders

Growing community of inventors

Philadelphia, PA, United States of America

Devin McDougald