Aav1 vectors and uses thereof for treatment of otic indications
AssigneeDecibel Therapeutics, Inc.
InventorsJoseph BURNS, Kathryn ELLIS, Matthew NGUYEN, Adam PALERMO, Gabriela PREGERNIG, Fuxin SHI, Jonathon WHITTON
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Abstract
The invention provides AAV1 vectors that can be used to transduce multiple inner ear cell types and their use for treatment of hearing loss, deafness, tinnitus, and vestibular dysfunction.
Claims14
A method of expressing a polynucleotide that encodes Gap Junction Protein Beta 6 (Gib6) operably linked to a promoter in developed inner ear cells in a human subject, the method comprising administering to the developed inner ear cells of the human subject a serotype 1 adeno-associated virus (AAV1) vector harvested from a cell lysate, the AAV1 vector comprising the polynucleotide, wherein the administering is in an amount that transduces one or more inner ear cell types selected from the group consisting of vestibular dark cells, Border cells, inner phalangeal cells, inner pillar cells, outer pillar cells, first row Deiters' cells, second row Deiters' cells, third row Deiters' cells, Hensen's cells, Claudius cells, interdental cells, basal cells of the stria vascularis, intermediate cells of the stria vascularis, marginal cells of the stria vascularis, and fibrocytes.
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A method of expressing a polynucleotide that encodes Solute Carrier Family 26 Member 4 (Slc26a4) operably linked to a promoter in developed inner ear cells in a human subject, the method comprising administering to the developed inner ear cells of the human subject an AAV1 vector harvested from a cell lysate, the AAV1 vector comprising the polynucleotide, wherein the administering is in an amount that transduces one or more inner ear cell types selected from the group consisting of spiral prominence cells, root cells, and vestibular dark cells.
Dependencies are inferred from the claim text.