Non-viral gene delivery in skeletal muscle: a protein factory

被引：161

作者：

Lu, QL ^{[1
]}

Bou-Gharios, G ^{[1
]}

Partridge, TA ^{[1
]}

机构：

[1] Univ London Imperial Coll Sci Technol & Med, Muscle Cell Biol Grp, MRC Clin Sci Ctr, Fac Med, London W12 0NN, England

来源：

GENE THERAPY | 2003年 / 10卷 / 02期

关键词：

plasmid DNA; skeletal muscle; gene delivery;

D O I：

10.1038/sj.gt.3301874

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

Ever since the publication of the first reports in 1990 using skeletal muscle as a direct target for expressing foreign transgenes, an avalanche of papers has identified a variety of proteins that can be synthesized and correctly processed by skeletal muscle. The impetus to the development of such applications is not only amelioration of muscle diseases, but also a range of therapeutic applications, from immunization to delivery of therapeutic proteins, such as clotting factors and hormones. Although the most efficient way of introducing transgenes into muscle fibres has been by a variety of recombinant viral vectors, there are potential benefits in the use of non-viral vectors. In this review we assess the recent advances in construction and delivery of naked plasmid DNA to skeletal muscle and highlight the options available for further improvements to raise efficiency to therapeutic levels.

引用

页码：131 / 142

页数：12

共 127 条

[1] Gene transfer into muscle by electroporation in vivo [J].