[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.
Petersen
Line Barrett
LB
Aarhus Universitet, Institut for Human Genetik, Denmark. line.barrett@studmed.au.dk
Jensen
Thomas G
TG
D015316:Genetic Therapy / Q000379:methods; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D016511:Severe Combined Immunodeficiency / Q000235:genetics / Q000628:therapy*; D016896:Treatment Outcome