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Gene therapy for severe combined immune deficiency

Published online by Cambridge University Press:  05 July 2004

Waseem Qasim ,
H. Bobby Gaspar  and
Adrian J. Thrasher
Waseem Qasim
Affiliation:
Molecular Immunology Unit, Institute of Child Health, 30 Guilford Street, London, WC1N 1EH, UK.
H. Bobby Gaspar
Affiliation:
Molecular Immunology Unit, Institute of Child Health, 30 Guilford Street, London, WC1N 1EH, UK.
Adrian J. Thrasher
Affiliation:
Molecular Immunology Unit, Institute of Child Health, 30 Guilford Street, London, WC1N 1EH, UK.
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Abstract

Infants born with severe combined immune deficiencies are prone to life-threatening infections and, without treatment, do not survive beyond the first year of life. Haematopoietic stem cell transplantation from a fully matched donor offers the possibility of cure. In the absence of a suitable matched donor, haploidentical transplants from a parental donor may be undertaken, but these are associated with more complications and lower success rates. Recently, an alternative therapeutic option based on retroviral gene delivery has been used to correct X-linked severe combined immune deficiency (SCID-X1) and adenosine deaminase deficiency. Clinical trials have established that in situations where ex vivo gene transfer into haematopoietic progenitor cells confers a strong selective advantage, the procedure is a feasible alternative to haploidentical transplantation, with favourable kinetics of immune reconstitution.

Keywords

severe combined immune deficiencySCIDgene therapyhaematopoietic stem cell transplantationHSCTretroviral vector
Type
Review Article
Information
Expert Reviews in Molecular Medicine , Volume 6 , Issue 4 , 2 July 2004 , pp. 1 - 15
Copyright
© Cambridge University Press 2004

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