Adeno-associated viral vectors for transduction of cochlea
AssigneeThe Children's Hospital of Philadelphia
InventorsBeverly DAVIDSON, Paul RANUM
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Abstract
Provided herein are compositions and methods for delivering a molecular therapeutic to the cochlea of a subject. The methods comprise administering an adeno-associated virus (AAV) to the cerebrospinal fluid of the subject. The AAVs encode a therapeutic transgene for molecular therapy. Optionally, the therapeutic transgene may be operably linked to a cochlea-specific promoter.
Claims67
A method to deliver a therapeutic transgene to the cochlea of a subject, comprising administering to the cerebrospinal fluid of the subject a modified adeno-associated virus (AAV) encoding a therapeutic transgene that treats or prevents a hearing or vestibular disorder when expressed in a cell of the cochlea.
A modified adeno-associated virus (AAV) comprising a therapeutic transgene operably linked to a cochlea-specific promoter.
Dependencies are inferred from the claim text.