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1. (WO2018204734) COMPOSITIONS AND METHODS FOR EXPRESSING OTOFERLIN

Pub. No.:    WO/2018/204734    International Application No.:    PCT/US2018/031009
Publication Date: Fri Nov 09 00:59:59 CET 2018 International Filing Date: Sat May 05 01:59:59 CEST 2018
IPC: C12N 5/079
C12N 15/86
Applicants: UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INCORPORATED
THE REGENTS OF THE UNIVERSITY OF CALIFORNIA
Inventors: BOYE, Sanford, L.
DYKA, Frank
HAUSWIRTH, William, W.
AKIL, Omar
Title: COMPOSITIONS AND METHODS FOR EXPRESSING OTOFERLIN
Abstract:
Provided herein are methods and compositions for expressing Otoferlin, e.g., utilizing adeno-associated viral (AAV) particles. Such methods and compositions may be useful for treatment of diseases such as Deafness, Autosomal Recessive 9 (DFNB9).