Gene therapy for immune disorders: good news tempered by bad news
- PMID: 16630946
- DOI: 10.1016/j.jaci.2006.01.041
Gene therapy for immune disorders: good news tempered by bad news
Abstract
After a dozen years of human gene therapy trials characterized by minimal gene correction and disappointing clinical impact, the field of gene therapy received some good news in 2000. Infants with X-linked severe combined immunodeficiency who received retroviral gene addition to cells from their bone marrow developed impressive immune reconstitution. During the following 2 years, additional patients were treated and the news was even better-babies receiving gene therapy had sustained T-cell production and in several cases developed better cell function than most patients treated with standard bone marrow transplants. Unfortunately, bad news followed. Three of the patients experienced leukemic T-cell expansions, found to be associated with retroviral insertions into genomic DNA. Where does the field stand today?
Comment in
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The last 80 years in primary immunodeficiency: how far have we come, how far need we go?J Allergy Clin Immunol. 2006 Apr;117(4):748-52. doi: 10.1016/j.jaci.2006.02.029. J Allergy Clin Immunol. 2006. PMID: 16630928 Review. No abstract available.
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