Aav vectors for treatment of dominant retinitis pigmentosa

JP2025016451AActive Publication Date: 2025-02-04UNIV OF FLORIDA RESEARCH FOUNDATION INC +1
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Patent Information

Application Number
JP2024168632
Authority / Receiving Office
JP · JP
Patent Type
Applications
Current Assignee / Owner
Priority Date
2016-09-22
Filing Date
2024-09-27
Publication Date
2025-02-04
Estimated Expiration
2037-03-01

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Abstract

To provide compositions and methods for treating retinitis pigmentosa (for example, dominant retinitis pigmentosa) in a subject.SOLUTION: The present invention 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 is also delivered to the subject.SELECTED DRAWING: None
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