Gene Therapy for Metabolic Diseases of the Liver

被引：0
作者：
Kittichai Promrat
George Y. Wu
Catherine H. Wu
机构：
[1] University of Connecticut Health Center,Department of Medicine, Division of Gastroenterology
[2] University of Connecticut Health Center,Hepatology
来源：
BioDrugs | 2000年 / 13卷
关键词：
Gene Therapy; Adenoviral Vector; Hammerhead Ribozyme; Nonviral Vector; Asialoglycoprotein Receptor;
D O I：
暂无
中图分类号：
学科分类号：
摘要：
Significant advances have been made in the field of liver-directed gene therapy. Many diseases are potential targets for gene therapy, including diseases that have exclusive liver involvement and those with systemic manifestations as a result of defective protein synthesis from the liver. Examples are Crigler-Najjar syndrome type 1, α1-antitrypsin deficiency and haemophilia A and B.
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页码：177 / 188
页数：11
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