Jean Bennett,Jeannette Bennicelli,Junwei Sun,Ji Yun Song,Sergei Nikonov
申请号：
US16489770
公开号：
US20190388561A1
申请日:
2018.03.01
申请国别(地区):
US
年份:
2019
代理人:
摘要：
Compositions and methods are provided for treating Leber congenital amaurosis (LCA) in a subject. In one aspect, a recombinant adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding Lebercilin. In another aspect, Lebercilin has an amino acid sequence of SEQ ID NO: 1. In yet another aspect, the nucleic acid molecule has a sequence of SEQ ID NO: 3 or a variant thereof. In desired embodiments, the subject is human, cat, dog, sheep, or non-human primate.
