[Gene therapy for severe combined immunodeficiency].
New results with gene therapy for severe combined immunodeficiency due to adenosine deaminase deficiency are promising. We review a clinical project in which ten children were treated with gene modified autologous haematopoietic stem cells. After treatment, eight patients were able to do without enzyme-replacement therapy, and nine patients showed improved immune function and sustained low concentration of toxic metabolites. No clonal outgrow was observed indicating a limited risk for future malignant development. Despite these promising results, the safety of gene therapy can still be improved.
Aarhus Universitet, Institut for Human Genetik, Denmark. line.barrett@studmed.au.dk
Line Barrett
LB
Petersen
Thomas G
TG
Jensen
D015316:Genetic Therapy / Q000379:methods; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D016511:Severe Combined Immunodeficiency / Q000235:genetics / Q000628:therapy*; D016896:Treatment Outcome