Materials and methods for delivering nucleic acids to cochlear and vestibular cells
AssigneesMassachusetts Eye and Ear Infirmary, The Schepens Eye Research Institute, Inc., Children’s Medical Center Corporation
InventorsKonstantina Stankovic, Luk H. Vandenberghe, Jeffrey Holt, Gwenaelle Geleoc
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Abstract
Provided herein are materials and methods for efficiently delivering nucleic acids to cochlear and vestibular cells.
Claims39
An AAV vector comprising an Anc80 capsid protein and one or more transgenes selected from the group consisting of TMC1, TMC2, MYO7A, USCH1C, CDH23, PCDH15, SANS, CIB2, USH2A, VLGR1, WHRN, CLRN1, PDZD7.
A method of delivering a transgene to one or more cells in the inner ear in a subject, the method comprising:
- administering an adeno-associated virus (AAV) to the inner ear in a subject, wherein the AAV comprises an Anc80 capsid protein and a transgene.
An article of manufacture comprising an AAV vector and a pharmaceutical composition, wherein the AAV vector comprises an Anc80 capsid protein and a transgene operably linked to a promoter.
A method of delivering a TMC1 or TMC2 transgene to one or more cells in the inner ear in a subject, the method comprising:
- administering an adeno-associated virus (AAV) to the inner ear in a subject, wherein the AAV comprises an Anc80 capsid protein and a transgene.
A method of delivering an Usher transgene to one or more cells in the inner ear in a subject, the method comprising:
- administering an adeno-associated virus (AAV) to the inner ear in a subject, wherein the AAV comprises an Anc80 capsid protein and a transgene.
Dependencies are inferred from the claim text.