Gene therapy improves immune function in preadolescents with X-linked severe combined immunodeficiency
Open Access
- 1 July 2007
- journal article
- clinical trial
- Published by American Society of Hematology in Blood
- Vol. 110 (1), 67-73
- https://doi.org/10.1182/blood-2006-11-058933
Abstract
Retroviral gene therapy can restore immunity to infants with X-linked severe combined immunodeficiency (XSCID) caused by mutations in the IL2RG gene encoding the common gamma chain (γc) of receptors for interleukins 2 (IL-2), −4, −7, −9, −15, and −21. We investigated the safety and efficacy of gene therapy as salvage treatment for older XSCID children with inadequate immune reconstitution despite prior bone marrow transplant from a parent. Subjects received retrovirus-transduced autologous peripherally mobilized CD34+ hematopoietic cells. T-cell function significantly improved in the youngest subject (age 10 years), and multilineage retroviral marking occurred in all 3 children.Keywords
This publication has 32 references indexed in Scilit:
- 660. The Effect of the B2 Mutation on Hematopoietic Reconstitution in the French X- SCID Gene Therapy TrialMolecular Therapy, 2006
- Clonal evidence for the transduction of CD34+ cells with lymphomyeloid differentiation potential and self-renewal capacity in the SCID-X1 gene therapy trialBlood, 2005
- Thyroid dysfunction after bone marrow transplantation for primary immunodeficiency without the use of total body irradiation in conditioningBone Marrow Transplantation, 2004
- LMO2 -Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1Science, 2003
- Transcription Start Regions in the Human Genome Are Favored Targets for MLV IntegrationScience, 2003
- Third-generation, self-inactivating gp91phoxlentivector corrects the oxidase defect in NOD/SCID mouse–repopulating peripheral blood–mobilized CD34+ cells from patients with X-linked chronic granulomatous diseaseBlood, 2002
- Gene Therapy of Human Severe Combined Immunodeficiency (SCID)-X1 DiseaseScience, 2000
- Dental manifestations in severe combined immunodeficiency following bone marrow transplantationBone Marrow Transplantation, 2000
- Use of Serum‐Free Medium with Fibronectin Fragment Enhanced Transduction in a System of Gas Permeable Plastic Containers to Achieve High Levels of Retrovirus Transduction at Clinical ScaleThe International Journal of Cell Cloning, 2000
- Final height and hormonal function after bone marrow transplantation in childrenThe Journal of Pediatrics, 1996