Occurrence of leukaemia following gene therapy of X-linked SCID Journal Article
| Authors: | Kohn, D. B.; Sadelain, M.; Glorioso, J. C. |
| Article Title: | Occurrence of leukaemia following gene therapy of X-linked SCID |
| Abstract: | Recombinant viral vectors have allowed gene transfer to be developed as a promising approach to the treatment of genetic diseases. Recently, gene therapy of children with X-linked severe combined immune deficiency resulted in impressive levels of immune reconstitution - a triumph that was later overshadowed by the development of leukaemia in two patients. What were the causes of this cancer, and how can the therapeutic benefits of gene therapy be achieved while minimizing risk to the patient? |
| Keywords: | leukemia; unclassified drug; genetics; clinical trial; review; risk benefit analysis; methodology; antineoplastic agent; cd40 ligand; protein; gene product; risk assessment; carcinogenesis; gene transfer; gene vector; genetic vectors; cell transformation, neoplastic; immunotherapy; cell transformation; janus kinase; gene therapy; beta thalassemia; virus recombinant; t cell leukemia; combined immunodeficiency; severe combined immunodeficiency; clinical trials; fanconi anemia; virus vector; beta globin; adrenoleukodystrophy; hurler syndrome; hemoglobinopathy; sickle cell anemia; wiskott aldrich syndrome protein; wiskott aldrich syndrome; glucose 6 phosphate dehydrogenase deficiency; glucose 6 phosphate dehydrogenase; adenosine deaminase; spectrin; cd18 antigen; metachromatic leukodystrophy; recombinant dna; gaucher disease; humans; human; priority journal; protein p47; insertional oncogenesis; cerebroside sulfatase; ferrochelatase; gamma c dna; glucosylceramidase; glycoprotein gp 91; janus 3 kinase; levo iduronidase; protein fanc a; protein fanc b; protein fanc c; protein fanc d; protein fanc e; agammaglobulinemia; chronic granulomatous disease; leukocyte adhesion deficiency; x chromosome linked disorder; genetic diseases, x-linked |
| Journal Title: | Nature Reviews Cancer |
| Volume: | 3 |
| Issue: | 7 |
| ISSN: | 1474-175X |
| Publisher: | Nature Publishing Group |
| Date Published: | 2003-07-01 |
| Start Page: | 477 |
| End Page: | 488 |
| Language: | English |
| PUBMED: | 12835668 |
| PROVIDER: | scopus |
| DOI: | 10.1038/nrc1122 |
| DOI/URL: | |
| Notes: | Export Date: 25 September 2014 -- Source: Scopus |
