Gene Therapy For Usher Syndrome Type 1B
Gene Therapy For Usher Syndrome Type 1B
A gene therapy that may prevent the blindness and correct the deafness associated with Usher 1B Syndrome, an inherited recessive loss-of-function disorder caused by mutations in the myosin VIIa gene.
San Diego, CA, United States
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Technology Description

UCSD researchers have discovered a gene therapy that may prevent the blindness and correct the deafness associated with Usher 1B Syndrome, an inherited recessive loss-of-function disorder caused by mutations in the myosin VIIa gene. These patients may be born deaf or develop deafness shortly thereafter, and later develop retinal degeneration (retinitis pigmentosa) in their teens. This method incorporates full-length human Myosin VIIA cDNA, as well as promoters to direct tissue-specific expression of the gene. 


Claims:
1. A method for the treatment or amelioration of an Usher 1B syndrome ocular disease, comprising delivering subretinally to target cells by injection in at least one eye of a subject in need of said treatment, an expression vehicle comprising or consisting of a human immunodeficiency virus-1 (HIV-1) vector comprising: (i) a CMV-MYO7A promoter or an equivalent promoter active in a photoreceptor cell and/or a retinal pigment epithelium (RPE) cell in operable linkage with a polynucleotide sequence encoding a myosin VIIA (MYO7A) protein, and (ii) a chromosomal integration sequence, or a chromosomal integration sequence and a chromatin insulator, wherein the MYO7A protein is expressed in said target cells, thereby treating the Usher 1B syndrome ocular disease in said subject. 
 

Applications

This technology represents the first therapeutic method available to treat and/or prevent this orphan disease, Usher Syndrome type 1B (approximately 4,000 cases in the US.) The gene can be delivered to cells in the eye to enable expression of the normal protein.

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