Children's Medical Center Corporation

Yukako Asai

Jeffrey Holt

Bernard Schneider

Gwenaelle Geleoc

Paola Andrea Solanes Vega

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.

Compositions and methods for delivering nucleic acids to cochlear and vestibular cells

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. Some embodiments are directed to a synthetic inner ear hair cell targeting adeno-associated virus (AAV) vector, a cell comprising the synthetic inner ear hair cell targeting AAV vector, and method of treating Usher Syndrome in a subject using the synthetic inner ear hair cell targeting AAV vector.

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Boston, MA, United States of America

Yukako Asai