Gene therapy for hearing loss and vestibular disorders
By using an AAV vector linked to a recombinant nucleic acid molecule and a TOMT regulatory element to specifically express relevant genes in cochlear and vestibular hair cells, the problems of non-target cell expression and toxicity in existing gene therapy methods have been solved, achieving efficient and sustained recovery of hearing and vestibular function.
Patent Information
- Authority / Receiving Office
- CN · China
- Patent Type
- Applications(China)
- Current Assignee / Owner
- UNIV OF PITTSBURGH OF THE COMMONWEALTH SYST OF HIGHER EDUCATION
- Filing Date
- 2024-10-18
- Publication Date
- 2026-07-14
AI Technical Summary
Existing gene therapy methods for treating hearing loss and vestibular disorders use pan-expression promoters that lead to abnormally high levels of gene expression in multiple cell types, resulting in partial functional recovery with unsustainable effects. Furthermore, existing viral vectors are toxic to hair cells.
By using recombinant nucleic acid molecules linked to transmembrane O-methyltransferase (TOMT) regulatory elements, genes related to hearing loss and vestibular disorders, such as the TOMT gene, are specifically expressed in cochlear and vestibular hair cells via adeno-associated virus (AAV) vectors, achieving efficient and safe expression of target cells.
This approach enables highly efficient and sustained gene therapy in the cochlea and vestibular hair cells, significantly salvaging hearing and vestibular function while avoiding the toxic effects on non-target cells.
Smart Images
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