Medicine. Gene therapy--new challenges ahead.
other · Level V
Where this comes from
- Record sourced from PubMed, PMID 14563994.
- No licence information is recorded for this record.
- Because redistribution is not established, this page shows the abstract only. Follow the links below for the full text.
Abstract
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.
Medical subject headings
- Genetic Therapy
- Leukemia-Lymphoma, Adult T-Cell
- Severe Combined Immunodeficiency