Gene Therapy--New Challenges Ahead
- 17 October 2003
- journal article
- editorial
- Published by American Association for the Advancement of Science (AAAS) in Science
- Vol. 302 (5644), 400-401
- https://doi.org/10.1126/science.1091258
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.Keywords
This publication has 9 references indexed in Scilit:
- LMO2 -Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1Science, 2003
- American society of gene therapy (ASGT) ad hoc subcommittee on retroviral-mediated gene transfer to hematopoietic stem cellsMolecular Therapy, 2003
- Transcription Start Regions in the Human Genome Are Favored Targets for MLV IntegrationScience, 2003
- Side effects of retroviral gene transfer into hematopoietic stem cellsBlood, 2003
- HIV-1 Integration in the Human Genome Favors Active Genes and Local HotspotsCell, 2002
- Correction of ADA-SCID by Stem Cell Gene Therapy Combined with Nonmyeloablative ConditioningScience, 2002
- Polyclonal long-term repopulating stem cell clones in a primate modelBlood, 2002
- Murine Leukemia Induced by Retroviral Gene MarkingScience, 2002
- Sustained Correction of X-Linked Severe Combined Immunodeficiency by ex Vivo Gene TherapyNew England Journal of Medicine, 2002