BENNETT, JEAN,BENNICELLI, JEANNETTE,SUN, JUNWEI,SONG, JI-YUN,NIKONOV, SERGEI
申请号：
CA3054136
公开号：
CA3054136A1
申请日:
2018.03.01
申请国别(地区):
CA
年份:
2018
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.
