| 摘要 |
<p>A method for the treatment of diseases associated with mutations in MY07A or CEP290 genes, especially the Usher Syndrome type IB and Leber congenital amaurosis, by administering to a subject in need thereof an adeno-associated viral vector encoding a MYO7A or a CEP290 protein; genetic constructs and adeno-associated viral vectors for use in this method.</p> |
