Review
Gene Therapy (2003) 10, 1999–2004. doi:10.1038/sj.gt.3302150
Gene therapy progress and prospects: gene therapy for severe combined immunodeficiency
H B Gaspar1, S Howe1 and A J Thrasher1
1Molecular Immunology Unit, Institute of Child Health, London, UK
Correspondence: Dr AJ Thrasher, Molecular Immunology Unit, Institute of Child Health, 30 Guilford Street, London WC1N 1EH, UK
Abstract
Severe combined immunodeficiencies have long been targeted as a group of disorders amenable to gene therapy because of their defined molecular biology and pathophysiology, and the prediction that corrected cells would have profound growth and survival advantage. Recently, several clinical studies have shown that conventional gene transfer technology can produce major beneficial therapeutic effects in these patients, but, as for all cellular and pharmacological treatment approaches, with a finite potential for toxicity.
Keywords:
SCID-X1, ADA, PEG-ADA, LMO-2, insertional mutagenesis