Purified hematopoietic stem cell transplantation: the next generation of blood and immune replacement.

Purified hematopoietic stem cell transplantation: the next generation of blood and immune replacement.
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DOI:
10.1016/j.iac.2010.03.003
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发表时间:
2010-05
影响因子:
2.6
通讯作者:
Weissman, Irving L.
Weissman, Irving L.
中科院分区:
医学3区
文献类型:
--
作者:
Czechowicz, Agnieszka;Weissman, Irving L.

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Severe Combined Immunodeficiency (SCID), Systemic Lupus Erythematosus (SLE), and Type I Diabetes share one commonality: these diverse disorders can all be attributed to faulty immune effector cells which are largely caused by genetic mutations that alter hematopoietic cell-intrinsic function. These defective immune cells inherit their genetic deficiencies from hematopoietic stem cells (HSC) as they differentiate. Thus, each of these unique diseases should be theoretically curable through the same strategy: replacement of patients’ HSCs carrying the problematic mutation with normal HSCs from disease-free donors, thereby generating entire new, healthy hematolymphoid systems. Replacement of disease-causing stem cells with healthy ones has been achieved clinically via hematopoietic cell transplantation (HCT) for the last 50 years, as a treatment modality for a variety of cancers and immunodeficiencies with moderate, but increasing success. This has traditionally included transplantation of mixed hematopoietic populations that include HSC and other cells, such as T-cells. This review article explores and delineates the potential expansion of this technique to treat a variety of inherited diseases of immune function, the current barriers in HCT and pure HSC transplantation, as well as the up-and-coming strategies to combat these obstacles.
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