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US 12,281,313 B2Granted
Compositions and methods for delivering nucleic acids to cochlear and vestibular cells
AssigneeChildren's Medical Center Corporation
InventorsJeffrey R. Holt, Yukako Asai, Paola Andrea Solanes Vega, Bernard Schneider
Life of this patent
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Abstract
Provided herein are materials and methods for efficiently delivering nucleic acids to cochlear and vestibular cells, and methods of treating sensory transduction disorders associated with a genetic defect.
Claims13
An AAV vector, wherein the vector comprises:
- a polynucleotide encoding:
- a capsid comprising amino acid sequence:
- TLAVPFK (SEQ ID NO:
- 27);
- and
- a polypeptide selected from the group consisting of TMC1, TMC2, MYO7A, USH1C, CDH23, PCDH15, SANS, CIB2, USH2A, VLGR1, WHRN, CLRN1, PDZD7, KCNQ4, TMPRSS3, STRC, EYA4, harmonin-a, b, and c, OTOF, GPR98, MYO6, MYO15A, LOXHD1, POU3F4, EYA1, WFS1, ACTG1, TMIE, PJVK, SYNE4, and FAM65B;
- and
- a promoter that directs expression of the polynucleotide encoding the polypeptide.
5 more elements
Dependencies are inferred from the claim text.