The promise of gene therapy for the treatment of α-1 antitrypsin deficiency

被引：4
作者：
Cruz, Pedro E.
Mueller, Christian
Flotte, Terence R. [1 ]
机构：
[1] Univ Florida, Div Nephrol, Coll Med, Gainesville, FL 32611 USA
[2] Univ Massachusetts, Sch Med, Worcester, MA 01655 USA
来源：
PHARMACOGENOMICS | 2007年 / 8卷 / 09期
关键词：
alpha-1; antitrypsin; AAT deficiency; gene therapy; Pim; Piz; siRNA; rAAV;
D O I：
10.2217/14622416.8.9.1191
中图分类号：
R9 [药学];
学科分类号：
1007 ;
摘要：
In the last 13 years, three gene therapy trials for the treatment of alpha-1 antitrypsin deficiency have been conducted. The first trial delivered plasmid encoding the (alpha-1 antitrypsin cDNA to the nasal epithelium using cationic liposomes. The last two trials delivered recombinant adeno-associated vectors encoding the a-1 antitrypsin cDNA by intramuscular injection. In this review, the progress of ongoing clinical trials and new gene therapy technologies is discussed.
引用
收藏
页码：1191 / 1198
页数：8
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