Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency

X-linked severe combined immunodeficiency (SCID-X1) is a profound deficiency of T, B, and natural killer (NK) cell immunity caused by mutations in IL2RG encoding the common chain (γc) of several interleukin receptors. Gamma-retroviral (γRV) gene therapy of SCID-X1 infants without conditioning restor...

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Published in:Sci Transl Med
Main Authors: De Ravin, Suk See, Wu, Xiaolin, Moir, Susan, Kardava, Lela, Anaya-O’Brien, Sandra, Kwatemaa, Nana, Littel, Patricia, Theobald, Narda, Choi, Uimook, Su, Ling, Marquesen, Martha, Hilligoss, Dianne, Lee, Janet, Buckner, Clarissa M., Zarember, Kol A., O’Connor, Geraldine, McVicar, Daniel, Kuhns, Douglas, Throm, Robert E., Zhou, Sheng, Notarangelo, Luigi D., Hanson, I. Celine, Cowan, Mort J., Kang, Elizabeth, Hadigan, Coleen, Meagher, Michael, Gray, John T., Sorrentino, Brian P., Malech, Harry L.
Format: Artigo
Language:Inglês
Published: 2016
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Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC5557273/
https://ncbi.nlm.nih.gov/pubmed/27099176
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/scitranslmed.aad8856
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