您的位置: 首页 > 农业专利 > 详情页

GENE THERAPY FOR OCULAR DISORDERS
专利权人:
The Trustees of the University of Pennsylvania
发明人:
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.
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/

意 见 箱

匿名:登录

个人用户登录

找回密码

第三方账号登录

忘记密码

个人用户注册

必须为有效邮箱
6~16位数字与字母组合
6~16位数字与字母组合
请输入正确的手机号码

信息补充