Article
Rescue of Hearing by Gene Delivery to Inner-Ear Hair Cells Using Exosome-Associated AAV.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Feb 2017
György Bence, Sage Cyrille, Indzhykulian Artur A, Scheffer Deborah I, Brisson Alain R, Tan Sisareuth, Wu Xudong, Volak Adrienn, Mu Dakai, Tamvakologos Panos I, Li Yaqiao, Fitzpatrick Zachary, Ericsson Maria, Breakefield Xandra O, Corey David P, Maguire Casey A
Abstract excerpt
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...
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