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

Gene Therapy volume 10, pages 131–142 (2003)Cite this article

Abstract

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.

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  1. Muscle Cell Biology Group, MRC Clinical Sciences Centre, Faculty of Medicine, Imperial College of Science, Technology and Medicine, London, UK

    Q L Lu, G Bou-Gharios & T A Partridge

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Lu, Q., Bou-Gharios, G. & Partridge, T. Non-viral gene delivery in skeletal muscle: a protein factory. Gene Ther 10, 131–142 (2003). https://doi.org/10.1038/sj.gt.3301874

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