Musings on genome medicine: gene therapy

被引：1
|
作者：
Nathan, David G. [1 ]
Orkin, Stuart H. [1 ]
机构：
[1] Dana Farber Canc Inst, Boston, MA 02115 USA
来源：
GENOME MEDICINE | 2009年 / 1卷
关键词：
Hematopoietic Stem Cell; Long Terminal Repeat; Chronic Granulomatous Disease; Adenosine Deaminase; Beta Globin Gene;
D O I：
10.1186/gm38
中图分类号：
Q3 [遗传学];
学科分类号：
071007 ; 090102 ;
摘要：
Though the field has moved with glacial speed, gene therapies have been carried out successfully in patients with bone marrow disorders including immune deficiencies. The field may be poised to move forward more rapidly, but many barriers have yet to be surmounted.
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页数：4
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