AAV vectors for treatment of dominant retinitis pigmentosa

US11118185B2 · kind B2 · utility

Assignees

• University of Florida Research Foundation, INC. · US
• The Trustees of the University of Pennsylvania · US

Inventors

• Alfred S. Lewin · Gainesville, US
• William W. Hauswirth · Gainesville, US
• Michael T. Massengill · Gainesville, US
• William A. Beltran · Philadelphia, US
• Gustavo D. Aguirre · Philadelphia, US
• Artur Cideciyan · Philadelphia, US
• Samuel G. Jacobson · Levittown, US

Key dates

• Filing date: Mar 1, 2017
• Grant date: Sep 14, 2021
• Expiry date: Jun 2, 2038

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2750/14143
• WIPO field: Biotechnology
• WIPO sector: Chemistry

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.