Aav capsid protein mutants targeting glial cells and uses thereof
By inserting peptides into the AAV-DJ capsid protein to design AAV capsid protein mutants that target glial cells, the problem of low transduction efficiency of existing AAV vectors in adult rat inner ear support cells was solved, achieving efficient transduction in both neonatal and adult rat inner ear support cells, thus improving the gene therapy effect for hereditary hearing loss.
Patent Information
- Authority / Receiving Office
- CN · China
- Patent Type
- Applications(China)
- Current Assignee / Owner
- EYE & ENT HOSPITAL SHANGHAI MEDICAL SCHOOL FUDAN UNIV
- Filing Date
- 2026-03-23
- Publication Date
- 2026-07-17
AI Technical Summary
Existing AAV vectors are difficult to efficiently target and transduce the supporting cells of the inner ear of adult mice, affecting the efficacy of gene therapy for hereditary hearing loss.
A mutant of AAV capsid protein targeting glial cells was designed by inserting a polypeptide with glial cell infection properties between N589 and R590 of the AAV-DJ capsid protein, thus constructing an AAV capsid protein mutant that can efficiently transduce supporting cells of the basilar membrane of the inner ear of newborn and adult mice.
This AAV capsid protein mutant significantly improves transduction efficiency in the inner ear support cells of newborn and adult mice, providing a more effective gene therapy tool, especially for hereditary hearing loss associated with gene mutations in support cells.
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