Gene therapy with viral vectors

被引：191

作者：

Kootstra, NA ^{[1
]}

Verma, IM ^{[1
]}

机构：

[1] Salk Inst Biol Studies, Genet Lab, La Jolla, CA 92037 USA

来源：

ANNUAL REVIEW OF PHARMACOLOGY AND TOXICOLOGY | 2003年 / 43卷

关键词：

AAV; retrovirus; lentivirus; adenovirus; HSV;

D O I：

10.1146/annurev.pharmtox.43.100901.140257

中图分类号：

R9 [药学];

学科分类号：

1007 ;

摘要：

A key factor in the success of gene therapy is the development of gene delivery systems that are capable of efficient gene transfer in a broad variety of tissues, without causing any pathogenic effect. Currently, viral vectors based on many different viruses have been developed, and their performance and pathogenicity has been evaluated in animal models. The results of these studies form the basis for the first clinical trials for correcting genetic disorders using retroviral, adenoviral, and adeno-associated viral vectors. Even though the results of these trials are encouraging, vector development is still required to improve and refine future treatment of hereditary disorders.

引用

页码：413 / 439

页数：27

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