Prospects for stem cell-based therapy

Prospects for stem cell-based therapy
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DOI:
10.1016/j.cell.2008.02.009
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发表时间:
2008-02-22
期刊:
影响因子:
64.5
通讯作者:
Scadden, David T.
Scadden, David T.
中科院分区:
生物学1区
文献类型:
--
作者:
Daley, George Q.;Scadden, David T.

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许多器官中驻留的体细胞干细胞负责组织的维持和修复。再生医学的目标是通过从外源性来源移植这些细胞或从药理学上激活内源性干细胞来利用这些细胞。对于由单个基因突变引起的疾病,治疗目标是使用经过改造的干细胞来纠正遗传缺陷的组织替代。然而,在基于多能性人类干细胞的治疗方法进入临床之前,必须克服许多技术障碍。
Resident pools of somatic stem cells in many organs are responsible for tissue maintenance and repair. The goal of regenerative medicine is to exploit these cells either by transplanting them from an exogenous source or by activating endogenous stem cells pharmacologically. For diseases caused by mutations in a single gene, the therapeutic goal is tissue replacement using stem cells engineered to correct the genetic defect. However, a number of technical hurdles must be overcome before therapies based on pluripotent human stem cells can enter the clinic.