Encouraging results of gene therapy for severe combined immunodeficiency


NB. Please note that if you are outside North America, the embargo for LANCET press material is 0001 hours UK Time Friday 17 December 2004.

A UK study in this week's issue of THE LANCET provides further evidence that gene therapy can be effective in creating a functional immune system for infants with severe combined immunodeficiency (SCID).

SCID is a genetic disease which prevents the immune system--particularly the white blood cells--from functioning. This leaves individuals at high risk of infection, as documented many years ago by the patient, David Vetter, who lived in a 'bubble' for 12 years to prevent infection from the external environment.

In the latest study, 4 children with SCID had stem cells removed from their own bone marrow; the relevant 'good' gene was identified from bone-marrow cells and spliced into a leukaemia virus. This virus was then introduced back into the patients where the gene was replicated. Almost normal immune functioning became established in all 4 patients after the procedure.

Lead investigator Adrian Thrasher (Institute of Child Health, London) comments: "At last follow up, all patients were at home in normal family and social environments, without restrictions on activities or exposure."

An accompanying commentary (p 2155) by Marina Cavazzana-Calvo and Alain Fischer (H˘pital Necker-Enfants Malades, Paris, France) discusses all clinical research done in this field: 'Remarkably, 17 of these 18 patients had their immunodeficiencies corrected with clear and sustained clinical benefits. The data show that the results of this gene-therapy strategy are reproducible.'

Source: Eurekalert & others

Last reviewed: By John M. Grohol, Psy.D. on 21 Feb 2009
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