GENE-THERAPY FOR METABOLIC DISORDERS

被引：93
作者：
KAY, MA
WOO, SLC
机构：
[1] HOWARD HUGHES MED INST,HOUSTON,TX 77030
[2] BAYLOR COLL MED,HOUSTON,TX 77030
来源：
TRENDS IN GENETICS | 1994年 / 10卷 / 07期
关键词：
D O I：
10.1016/0168-9525(94)90173-2
中图分类号：
Q3 [遗传学];
学科分类号：
071007 ; 090102 ;
摘要：
Most diseases caused by genetic deficiencies could, in theory, be treated by the introduction and expression of a normal gene into an appropriate target tissue. It seems likely that gene therapy strategies for most metabolic disorders will not require strict gene regulation, as a fraction of the normal levels of gene activity could result in amelioration or significant improvement in the clinical outcome. Gene therapy is making rapid progress towards the goal of treating various disorders: here, we summarize the state of gene therapy for metabolic disorders.
引用
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页码：253 / 257
页数：5
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