- 专利标题： Globin gene therapy for treating hemoglobinopathies
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申请号： US15449416申请日： 2017-03-03
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公开(公告)号： US11717579B2公开(公告)日： 2023-08-08
- 发明人: Michel Sadelain , Isabelle Riviere , Jorge Mansilla-Soto , Xiuyan Wang , George Stamatoyannopoulos , John Stamatoyannopoulos , Mingdong Liu
- 申请人： MEMORIAL SLOAN-KETTERING CANCER CENTER , UNIVERSITY OF WASHINGTON
- 申请人地址： US NY New York
- 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： US NY New York; US WA Seattle
- 代理机构： Baker Botts L.L.P.
- 主分类号： C12N15/86
- IPC分类号： C12N15/86 ; C12N15/63 ; A61K48/00 ; C12N15/11 ; C07K14/805 ; C12N9/22 ; C12N15/85
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
- US20170173185A1 GLOBIN GENE THERAPY FOR TREATING HEMOGLOBINOPATHIES 公开/授权日：2017-06-22
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