Medicine. Gene therapy--new challenges ahead.
The successful use of retroviral gene transfer to treat 10 patients with X-linked severe combined immunodeficiency (SCID-X1) has been welcomed as evidence of the therapeutic potential of gene therapy. However, as Williams and Baum suggest in their Perspective, the discovery that 2 of the 10 patients developed leukemia within 3 years of gene therapy (Hacein-Bey-Abina et al.) reinforces the need to develop even more specific gene therapy interventions.
Williams
David A
DA
Division of Experimental Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA. david.williams@cchmc.org
Baum
Christopher
C
D048868:Adaptor Proteins, Signal Transducing; D002986:Clinical Trials as Topic; D004268:DNA-Binding Proteins / Q000235:genetics; D018014:Gene Transfer Techniques; D016147:Genes, Tumor Suppressor; D015316:Genetic Therapy / Q000009:adverse effects; D005822:Genetic Vectors; D018380:Hematopoietic Stem Cell Transplantation; D006412:Hematopoietic Stem Cells / Q000502:physiology; D006801:Humans; D007223:Infant; D060588:LIM Domain Proteins; D015459:Leukemia-Lymphoma, Adult T-Cell / Q000209:etiology*; D008667:Metalloproteins / Q000235:genetics; D016254:Mutagenesis, Insertional; D011518:Proto-Oncogene Proteins; D011519:Proto-Oncogenes; D015375:Receptors, Interleukin-2 / Q000235:genetics; D012190:Retroviridae / Q000235:genetics; D012307:Risk Factors; D016511:Severe Combined Immunodeficiency / Q000628:therapy*; D013601:T-Lymphocytes / Q000502:physiology; D019076:Transgenes
D048868:Adaptor Proteins, Signal Transducing; D004268:DNA-Binding Proteins; D060588:LIM Domain Proteins; C085117:LMO2 protein, human; D008667:Metalloproteins; D011518:Proto-Oncogene Proteins; D015375:Receptors, Interleukin-2