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1. (WO2018200542) GENE THERAPY FOR OCULAR DISORDERS

Pub. No.:    WO/2018/200542    International Application No.:    PCT/US2018/029167
Publication Date: Fri Nov 02 00:59:59 CET 2018 International Filing Date: Wed Apr 25 01:59:59 CEST 2018
IPC: C07K 14/47
C12N 15/86
C12N 9/80
C12N 7/00
A61K 9/00
A61K 48/00
A61K 38/17
Applicants: THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
Inventors: BENNETT, Jean
SUN, Junwei
SHINDLER, Kenneth
MCDOUGALD, Devin
Title: GENE THERAPY FOR OCULAR DISORDERS
Abstract:
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.