- 专利标题: Methods and compositions for treating chronic granulomatous disease
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申请号: US16329915申请日: 2017-09-01
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公开(公告)号: US11738053B2公开(公告)日: 2023-08-29
- 发明人: Senlin Li , Robert A Clark , Cang Chen , Yang Li
- 申请人: THE BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM
- 申请人地址: US TX Austin
- 专利权人: Board of Regents, The University of Texas System
- 当前专利权人: Board of Regents, The University of Texas System
- 当前专利权人地址: US TX Austin
- 国际申请: PCT/US2017/049815 2017.09.01
- 国际公布: WO2018/045266A 2018.03.08
- 进入国家日期: 2019-03-01
- 主分类号: A01N63/00
- IPC分类号: A01N63/00 ; C12N15/63 ; C07H21/04 ; A61K35/28 ; A61K38/19 ; A01K67/027 ; C12N15/86
摘要:
The invention relates to a method of treating chronic granulomatous disease through Hematopoietic stem cell (HSC) transplantation. The method comprises the steps of administering stem cell mobilization agent to human such that the target stem cell population migrates from host niches into the subject's blood. The target stem cells are removed from blood and administering the therapeutic stem cells to human and said therapeutic stem cells are engineered to express gp91phox. The steps are repeated multiple times i.e. at least four times. The mobilization agents used in the invention are granulocyte-colony stimulating factor and AMD3100. The method of HSC transplantation is effective in treatment of chronic granulomatous disease.
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