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Biotechnology and Applied Biochemistry (2003) 37, (219–223) (Printed in Great Britain)

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Perspectives on plasmid-based gene therapy: challenges for the product and the process

William J. Kelly¹

Chemical Engineering Department, Villanova University, 318 White Hall, 800 Lancaster Ave, Villanova, PA 19085, U.S.A.

Key words: DNA plasmids, Escherichia coli, gene delivery, gene therapy, plasmid purification, supercoiled DNA.

Abbreviations used: CTAB, cetyltrimethylammonium bromide; EU, endotoxin unit; AEX, anion-exchange chromatography.

¹e-mail william.j.kelly@villanova.edu.

Clinical trials for gene-therapy products began in the 1990s. Currently, most of the 630 completed or on-going trials are in Phase I and II. Because of the safety issues surrounding the use of viral vectors, approx. 25% of the ongoing clinical trials use DNA plasmids to carry the corrective gene (http://www.wiley.co.uk/genmed). The purpose of this perspective (and this issue) is to highlight some of the unique technical challenges and potential solutions associated with the manufacture and expression of a plasmid-based gene-therapy product.

Received 20 February 2003/27 March 2003; accepted 3 April 2003

Published as Immediate Publication 3 April 2003, DOI 10.1042/BA20030033

Portland Press Ltd ©2003