University of Florida Research Foundation Inc

University of Pennsylvania

Artur Cideciyan

William W Hauswirth

Alfred Samuel Lewin

Gustavo D Aguirre

William Beltran

Samuel Jacobson

Michael T Massengill

Aspects of the disclosure relate to methods and compositions useful for treating retinitis pigmentosa. In some aspects, the disclosure provides compositions and methods for delivering 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 a subject. In some embodiments, a replacement RHO coding sequence that is resistant to the interfering RNA also is delivered to the subject.

Compositions and methods for treatment of dominant retinitis pigmentosa

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Lafayette Hill, PA, United States of America

Artur Cideciyan