Gene therapy targeting cochlear cells
AssigneeResearch Institute At Nationwide Children's Hospital
InventorsKathrin Christine Meyer, Karim Bey
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Abstract
The present disclosure relates to methods of targeting specific cell types within the cochlea using optimized gene therapy vectors. In particular, the disclosure provides gene therapy vectors to specifically target cochlear cells and methods of treating hearing impairment and hearing-loss related disorders.
Claims6
A method of delivering a transgene to an inner hair cell in the cochlea of a subject, the method comprising administering a recombinant adeno-associated virus 9 (rAAV9) vector encoding the transgene, wherein the rAAV9 vector is administered to the subject using intrathecal delivery, and the transgene is delivered to an inner hair cell in the cochlea of the subject.
Dependencies are inferred from the claim text.