AAV vector for the treatment of retinitis dominant

By using interfering RNA and a recombinant adeno-associated virus vector to silence and replace mutated rhodopsin genes, the method addresses the lack of therapies for adRP, offering a potential treatment for restoring vision.

JP7872961B2Active Publication Date: 2026-06-11UNIV OF FLORIDA RESEARCH FOUNDATION INC +1
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Patent Information

Application Number
JP2024168632
Authority / Receiving Office
JP · JP
Patent Type
Patents
Current Assignee / Owner
Priority Date
2016-09-22
Filing Date
2024-09-27
Publication Date
2026-06-11
Estimated Expiration
2037-03-01

AI Technical Summary

Technical Problem

There are no approved drug or gene therapies for autosomal dominant retinitis pigmentosa (adRP), a disease that leads to blindness due to mutations in the rhodopsin gene, affecting approximately 1 in 12,000 people.

Method used

Administering an interfering RNA molecule, such as small interfering RNA (siRNA) or artificial microRNA (miRNA), to silence one or both alleles of the rhodopsin gene, combined with a recombinant adeno-associated virus (rAAV) vector to deliver a substituted rho gene that is resistant to the interfering RNA, thereby restoring photoreceptor cell function.

Benefits of technology

The approach effectively reduces endogenous rhodopsin expression and replaces it with a functional, resistant rho gene, potentially restoring or maintaining partial vision in subjects with adRP.

✦ Generated by Eureka AI based on patent content.

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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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Citation Information

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