- 专利标题： GLOBIN GENE THERAPY FOR TREATING HEMOGLOBINOPATHIES
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申请号： EP19185730.9申请日： 2015-09-04
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公开(公告)号： EP3620521A1公开(公告)日： 2020-03-11
- 发明人: SADELAIN, Michel , RIVIERE, Isabelle , MANSILLA-SOTO, Jorge , WANG, Xiuyan , STAMATOYANNOPOULOS, George , STAMATOYANNOPOULOS, John , LIU, Mingdong
- 申请人： Memorial Sloan-Kettering Cancer Center , University of Washington
- 申请人地址： 1275 York Avenue Box 524 New York, NY 10065 US
- 专利权人： Memorial Sloan-Kettering Cancer Center,University of Washington
- 当前专利权人： Memorial Sloan-Kettering Cancer Center,University of Washington
- 当前专利权人地址： 1275 York Avenue Box 524 New York, NY 10065 US
- 代理机构： Barker Brettell LLP
- 优先权： US201462045997P 20140904
- 主分类号： C12N15/113
- IPC分类号： C12N15/113 ; A61K48/00 ; C12N15/85 ; C12N15/867
摘要：
The presently disclosed subject matter provides for expression cassettes that allow for expression of a globin gene or a functional portion thereof, vectors comprising thereof, and cells transduced with such expression cassettes and vectors. The presently disclosed subject matter further provides methods for treating a hemoglobinopathy in a subject comprising administering an effective amount of such transduced cells to the subject.
公开/授权文献
- EP3620521B1 GLOBIN GENE THERAPY FOR TREATING HEMOGLOBINOPATHIES 公开/授权日：2024-05-29
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