University of Florida Research Foundation, Incorporated

University of Pennsylvania

Michael T Massengill

William W Hauswirth

Alfred Samuel Lewin

Gustavo D Aguirre

William Beltran

Samuel Jacobson

Artur V Cideciyan

Artur Cideciyan

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

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.

AAV vectors for treatment of dominant retinitis pigmentosa

Growing community of inventors

Gainesville, FL, United States of America

Michael T Massengill