Ontology highlight
ABSTRACT:
SUBMITTER: Gyorgy B
PROVIDER: S-EPMC5368844 | biostudies-literature | 2017 Feb
REPOSITORIES: biostudies-literature
Molecular therapy : the journal of the American Society of Gene Therapy 20170109 2
Adeno-associated virus (AAV) is a safe and effective vector for gene therapy for retinal disorders. Gene therapy for hearing disorders is not as advanced, in part because gene delivery to sensory hair cells of the inner ear is inefficient. Although AAV transduces the inner hair cells of the mouse cochlea, outer hair cells remain refractory to transduction. Here, we demonstrate that a vector, exosome-associated AAV (exo-AAV), is a potent carrier of transgenes to all inner ear hair cells. Exo-AAV1 ...[more]