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- Suk See De Ravin, Xiaolin Wu, Susan Moir, Sandra Anaya-O'Brien, Nana Kwatemaa, Patricia Littel, Narda Theobald, Uimook Choi, Ling Su, Martha Marquesen, Dianne Hilligoss, Janet Lee, Clarissa M Buckner, Kol A Zarember, Geraldine O'Connor, Daniel McVicar, Douglas Kuhns, Robert E Throm, Sheng Zhou, Luigi D Notarangelo, I Celine Hanson, Mort J Cowan, Elizabeth Kang, Coleen Hadigan, Michael Meagher, John T Gray, Brian P Sorrentino, Harry L Malech, and KardavaLela.
- Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH), Bethesda, MD 20892, USA. sderavin@niaid.nih.gov hmalech@niaid.nih.gov.
- Sci Transl Med. 2016 Apr 20; 8 (335): 335ra57.
AbstractX-linked severe combined immunodeficiency (SCID-X1) is a profound deficiency of T, B, and natural killer (NK) cell immunity caused by mutations inIL2RGencoding the common chain (γc) of several interleukin receptors. Gamma-retroviral (γRV) gene therapy of SCID-X1 infants without conditioning restores T cell immunity without B or NK cell correction, but similar treatment fails in older SCID-X1 children. We used a lentiviral gene therapy approach to treat five SCID-X1 patients with persistent immune dysfunction despite haploidentical hematopoietic stem cell (HSC) transplant in infancy. Follow-up data from two older patients demonstrate that lentiviral vector γc transduced autologous HSC gene therapy after nonmyeloablative busulfan conditioning achieves selective expansion of gene-marked T, NK, and B cells, which is associated with sustained restoration of humoral responses to immunization and clinical improvement at 2 to 3 years after treatment. Similar gene marking levels have been achieved in three younger patients, albeit with only 6 to 9 months of follow-up. Lentiviral gene therapy with reduced-intensity conditioning appears safe and can restore humoral immune function to posthaploidentical transplant older patients with SCID-X1.Copyright © 2016, American Association for the Advancement of Science.
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