Article
Lymphoid regeneration from gene-corrected SCID-X1 subject-derived iPSCs.
Cell stem cell - 2 Apr 2015
Menon Tushar, Firth Amy L, Scripture-Adams Deirdre D, Galic Zoran, Qualls Susan J, Gilmore William B, Ke Eugene, Singer Oded, Anderson Leif S, Bornzin Alexander R, Alexander Ian E, Zack Jerome A, Verma Inder M
Abstract excerpt
X-linked Severe Combined Immunodeficiency (SCID-X1) is a genetic disease that leaves newborns at high risk of serious infection and a predicted life span of less than 1 year in the absence of a matched bone marrow donor. The disease pathogenesis is due to mutations in the gene encoding the Interleukin-2 receptor gamma chain (IL-2Rγ), leading to a lack of functional lymphocytes. With the leukemogenic concerns of...
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