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Gene therapy in clinical medicine

Correspondence to:
Correspondence to:
Dr S Selkirk
Department of Neurology, University Hospital of Cleveland, Hanna House 5, 11100 Euclid Avenue, Cleveland, OH 44106, USA; stephen_selkirk{at}hotmail.com

Although the field of gene therapy has experienced significant setbacks and limited success, it is one of the most promising and active research fields in medicine. Interest in this therapeutic modality is based on the potential for treatment and cure of some of the most malignant and devastating diseases affecting humans. Over the next decade, the relevance of gene therapy to medical practices will increase and it will become important for physicians to understand the basic principles and strategies that underlie the therapeutic intervention. This report reviews the history, basic strategies, tools, and several current clinical paradigms for application.

Abbreviations: AAV, adenoassociated virus; AD, Alzheimer’s disease; BMD, Becker’s muscular dystrophy; CABG, coronary artery bypass; CAD, coronary artery disease; CF, cystic fibrosis; CFTR, cystic fibrosis transmembrane regulator; CNS, central nervous system; DMD, Duchenne muscular dystrophy; HSV, herpes simplex virus; MI, myocardial infarction; MMLV, mouse Moloney leukaemia virus; NGF, nerve growth factor; PVD, peripheral vascular disease; shRNA, short hairpin RNA; siRNA, short interfering RNA; VEGF, vascular endothelial growth factor

Keywords: gene therapy; cell transplantation; molecular biology; surrogate cell; viral vectors

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