Success for gene therapy: render unto Caesar that which is Caesar's.
Reports that two young children developed leukemia after being treated for immunodeficiency with their own retrovirally modified bone-marrow cells delivered a severe blow to confidence in gene therapy as a treatment. Two reports, published since the trial was initiated, now take away some of the mystery as to why these events happened and allay fears for the safety of gene therapy across all therapeutic applications.
Qiao
Jian
J
Molecular Medicine Program and Department of Immunology, Guggenheim 18, Mayo Foundation, 200 First Street SW, Rochester, MN 55905, USA.
Diaz
Rosa Maria
RM
Vile
Richard G
RG
D048868:Adaptor Proteins, Signal Transducing; D000818:Animals; D002648:Child; D002986:Clinical Trials as Topic / Q000592:standards* / Q000639:trends; D004268:DNA-Binding Proteins / Q000235:genetics; D015316:Genetic Therapy / Q000009:adverse effects* / Q000639:trends; D006801:Humans; D060588:LIM Domain Proteins; D007938:Leukemia / Q000209:etiology* / Q000235:genetics*; D008667:Metalloproteins / Q000235:genetics; D051379:Mice; D016254:Mutagenesis, Insertional / Q000235:genetics; D011518:Proto-Oncogene Proteins; D015375:Receptors, Interleukin-2 / Q000235:genetics; D016511:Severe Combined Immunodeficiency / Q000235:genetics* / Q000628:therapy*
D048868:Adaptor Proteins, Signal Transducing; D004268:DNA-Binding Proteins; D060588:LIM Domain Proteins; C085117:LMO2 protein, human; C513188:Lmo2 protein, mouse; D008667:Metalloproteins; D011518:Proto-Oncogene Proteins; D015375:Receptors, Interleukin-2