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|1. (WO2017151823) AAV VECTORS FOR TREATMENT OF DOMINANT RETINITIS PIGMENTOSA|
|Applicants:||UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INC.
THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
|Inventors:||LEWIN, Alfred, S.
HAUSWIRTH, William, W.
MASSENGILL, Michael, T.
AGUIRRE, Gustavo, D.
|Title:||AAV VECTORS FOR TREATMENT OF DOMINANT RETINITIS PIGMENTOSA|
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.