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16. Exosome-Associated AAV as a Novel Platform for Gene Therapy of Hearing Loss

Year: 2015
Type: article
Abstract: Introduction: In recent gene therapy trials, adeno-associated virus (AAV) vectors for diseases such as blindness and hemophilia were found to be safe and effective. Gene therapy for hearing and balance disorders is not as advanced, because gene delivery into the cochlea (particularly to sensory hair cells) is generally inefficient. Here we show that exosome-associated AAV vectors (vexosomes) are highly effective carriers of transgenes to hair cells.
DOI: 10.1016/s1525-0016(16)33620-6
Authors: Bence Gyorgy, Cyrille Sage, Deborah Scheffer, Artur A. Indzhykulian, Dakai Mu, Xandra O. Breakefield, Casey A. Maguire, David P. Corey
Language: English
Subfield: Genetics
Open Access status: hybrid
Estimated APC paid: $4,500
OpenAlex ID: W2562514873
FWCI:
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Related to: 10