- 专利标题： METHODS OF TREATING HUMAN X-LINKED RETINOSCHISIS USING GENE THERAPY
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申请号： PCT/US2022/079195申请日： 2022-11-03
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公开(公告)号： WO2023081739A1公开(公告)日： 2023-05-11
- 发明人: MU, Xueyan Peter
- 申请人： TEAMEDON INTERNATIONAL, INC.
- 申请人地址： 2275 Research Blvd, Suite 500
- 专利权人： TEAMEDON INTERNATIONAL, INC.
- 当前专利权人： TEAMEDON INTERNATIONAL, INC.
- 当前专利权人地址： 2275 Research Blvd, Suite 500
- 代理机构： SUN-HOFFMAN, Lin
- 优先权： US63/275,351 2021-11-03
- 主分类号： A61P27/02
- IPC分类号： A61P27/02 ; C12N15/86 ; A61P27/00
摘要：
A method of treating X-linked juvenile retinoschisis (XLRS) in a human subject includes subretinally delivering to the human subject a therapeutically effective amount of an rAAV vector. The rAAV vector includes a nucleic acid sequence comprising coding sequence for human RS1 protein. The rAAV vector can further include a mutated AAV2 VP3 capsid protein having a phenylalanine (F) for tyrosine (Y) substitution at each of the positions corresponding to Y444, Y500 and Y730 in a wild type AAV2 VP3 capsid protein.
