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

Authority / Receiving Office
JP · JP
Patent Type
Patents
Current Assignee / Owner
UNIV OF FLORIDA RESEARCH FOUNDATION INC
Filing Date
2024-09-27
Publication Date
2026-06-11

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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