Patent Number: 7,786,091

Title: Compositions and methods for ameliorating myosin VIIa defects

Abstract: 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.

Inventors: Williams; David (San Diego, CA), Yang; Xian-Jie (Culver City, CA)

Assignee: The Regents of the University of California

International Classification: A01N 43/04 (20060101); A61K 31/715 (20060101)

Expiration Date: 8/31/12018