University of California

Xian-Jie Yang

David L Williams

The invention provides compositions and methods for ameliorating defects in myosin VIIa (MYO7A) expression and/or function, including providing vectors for myosin VIIa expression and formulations comprising them, and methods of using them, for treating human retinitis pigmentosa (or retinal degeneration), and blindness and deafness such as that found in Usher syndrome. The invention provides in vivo gene therapy for ameliorating defects in myosin VIIa (MYO7A) expression and/or function, including compositions and methods for gene transfer of the human myosin VIIa (MYO7A) gene (the MYO7A gene.

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Culver City, CA, United States of America

Xian-Jie Yang