Harvard College

The General Hospital Corporation

The Ohio State University

Massachusetts Eye and Ear Infirmary

David P Corey

Casey Maguire

Maryna V Ivanchenko

Killian S Hanlon

Bence Gyorgy

Artur Indzhykulian

Cole WD Peters

Aspects of the disclosure relate to compositions and methods for treating hereditary hearing loss and/or vision loss, for example, due to Usher syndrome, Type 3A. In some embodiments, the disclosure provides a recombinant adeno-associated virus comprising: (i) an AAV-S capsid protein, and (ii) an isolated nucleic acid comprising a transgene (e.g., a transgene for expressing a clarin-1 protein). The present disclosure also provides methods of treating hereditary hearing loss and/or vision loss (e.g., Usher Syndrome, Type 3A) using the same.

Recombinant adeno associated virus encoding clarin-1 and uses thereof

Aspects of the disclosure relate to compositions and methods useful for treating hereditary hearing loss, for example, Usher syndrome type 3A or nonsyndromic hearing loss and deafness (DFNB1). In some embodiments, the disclosure provides isolated nucleic acids, vectors, and rAAV.9.PHP.B comprising a transgene encoding a Clarin-1 or a GJB2, and methods of treating hearing loss using the same.

AAV vectors encoding clarin-1 or GJB2 and uses thereof

Growing community of inventors

Cambridge, MA, United States of America

David P Corey