GENE THERAPIES FOR USHER SYNDROME (USH1B)

摘要

Aspects of the disclosure relate to compositions and methods useful for delivering minigenes to a subject. Accordingly, the disclosure is based, in part, on isolated nucleic acids and gene therapy vectors, such as viral (e.g., rAAV) vectors, comprising one or more gene fragments encoding a therapeutic gene product, such as a protein or peptide (e.g, a minigene). In some embodiments, the disclosure relates to gene therapy vectors encoding a USH1B protein (e.g, the gene product of USH1B, also referred to as MY 07 A)or a portion thereof. In some embodiments, compositions described by the disclosure are useful for treating diseases associated with mutations in the USH1B (MY07A) gene, for example Usher Syndrome.