Gene therapy for immune disorders: good news tempered by bad news

The Journal of Allergy and Clinical Immunology
Jennifer M Puck, H L Malech

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?

Citations

Sep 29, 2009·Expert Opinion on Biological Therapy·Sergey S Seregin, Andrea Amalfitano
Oct 13, 2007·The Journal of Allergy and Clinical Immunology·William T ShearerJennifer M Puck
Dec 28, 2007·Advances in Dermatology·Melissa Abrams, Amy Paller
Jun 28, 2007·Allergologia et immunopathologia·P Soler-PalacínT Español
Feb 23, 2007·Immunology·Danielle AwDonald B Palmer
Nov 25, 2006·Hematology·Peter E Newburger
Dec 13, 2006·Clinical & Developmental Immunology·Arvind KumarM Eric Gershwin

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