Hematopoietic stem cell gene therapy: selecting only the best

被引：19

作者：

Bank, A

机构：

[1] Columbia Univ Coll Phys & Surg, Armand HHSC, Dept Med, New York, NY 10032 USA

[2] Columbia Univ Coll Phys & Surg, Armand HHSC, Dept Genet & Dev, New York, NY 10032 USA

来源：

JOURNAL OF CLINICAL INVESTIGATION | 2003年 / 112卷 / 10期

关键词：

D O I：

10.1172/JCI200320336

中图分类号：

R-3 [医学研究方法]; R3 [基础医学];

学科分类号：

1001 ;

摘要：

Hematopoietic stem cell (HSC) gene therapy can potentially cure a variety of human hematopoietic diseases, such as sickle cell disease. Selection and expansion of gene-corrected HSCs has now been accomplished for the first time using HSC from large animals - dogs and humans - with a novel drug-resistance gene, MGMT, which is not expressed in normal HSCs (see the related articles beginning on pages 1561 and 1581). Highly efficient lentiviral transfer and expression of MGMT into relatively few HSCs led to repopulation of most of the hematopoietic compartment with gene-corrected cells following suitable drug treatment. This selection system may be useful in human clinical trials to permit gene therapy in autologous and allogeneic bone marrow transplantation settings.

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页码：1478 / 1480

页数：3

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