Patent ID: 7786091
Filing Date: 2010-08-31
Classification: A61K,A61P,C12N

Abstract:
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.