Hematopoietic stem cell gene therapy

被引：23
作者：
Emery, DW
Nishino, T
Murata, K
Fragkos, M
Stamatoyannopoulos, G
机构：
[1] Univ Washington, Dept Med, Div Med Genet, Seattle, WA 98195 USA
[2] Univ Crete, Sch Med, Dept Basic Sci, Iraklion, Crete, Greece
[3] Univ Crete, Inst Mol Biol & Biotechnol, Dept Basic Sci, Iraklion, Crete, Greece
来源：
INTERNATIONAL JOURNAL OF HEMATOLOGY | 2002年 / 75卷 / 03期
关键词：
gene therapy; hematopoiesis; stem cells; virus vectors;
D O I：
10.1007/BF02982035
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
Gene therapy applications that target hematopoietic stem cells (HSCs) offer great potential for the treatment of hematologic disease. Despite this promise, clinical success has been limited by poor rates of gene transfer, poor engraftment of modified cells, and poor levels of gene expression. We describe here the basic approach used for HSC gene therapy, briefly review some of the seminal clinical trials in the field, and describe several recent advances directed toward overcoming these limitations. (C)2002 The Japanese Society of Hematology.
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收藏
页码：228 / 236
页数：9
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