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1. (WO2017151823) AAV VECTORS FOR TREATMENT OF DOMINANT RETINITIS PIGMENTOSA

Pub. No.:    WO/2017/151823    International Application No.:    PCT/US2017/020289
Publication Date: Sat Sep 09 01:59:59 CEST 2017 International Filing Date: Thu Mar 02 00:59:59 CET 2017
IPC: C12N 15/79
C12N 15/86
C12N 15/85
C12N 15/10
C12N 15/11
C12N 15/113
C07H 21/02
Applicants: UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INC.
THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
Inventors: LEWIN, Alfred, S.
HAUSWIRTH, William, W.
MASSENGILL, Michael, T.
BELTRAN, William
AGUIRRE, Gustavo, D.
CIDECIYAN, Artur
JACOBSON, Samuel
Title: AAV VECTORS FOR TREATMENT OF DOMINANT RETINITIS PIGMENTOSA
Abstract:
Aspects of the disclosure relate to methods and compositions for treating retinitis pigmentosa. In some aspects, the disclosure provides compositions and methods for delivering an interfering nucleic acid (for example an interfering RNA) to a subject in order to reduce expression of one or both alleles of an endogenous rho gene (for example a mutant rho allele associated with retinitis pigmentosa) in the subject. In some embodiments, a replacement rho gene that is resistant to the interfering nucleic acid also is delivered to the subject.