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 Blood, 1 July 2007, Vol. 110, No. 1, pp. 67-73.
Prepublished online as a Blood First Edition Paper on March 19, 2007; DOI 10.1182/blood-2006-11-058933.

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GENE THERAPY

Gene therapy improves immune function in preadolescents with X-linked severe combined immunodeficiency

Javier Chinen1, Joie Davis1, Suk See De Ravin2, Beverly N. Hay1, Amy P. Hsu1, Gilda F. Linton2, Nora Naumann2, Effie Y. H. Nomicos2, Christopher Silvin1, Jean Ulrick2, Narda L. Whiting-Theobald2, Harry L. Malech2, and Jennifer M. Puck1

1 Genetics and Molecular Biology Branch, National Human Genome Research Institute, and 2 Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Department of Health and Human Services (DHHS), Bethesda, MD

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 ({gamma}c) 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.


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Gene therapy as salvage
Donald B. Kohn
Blood 2007 110: 4. [Full Text] [PDF]



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