Gene Correction of iPSCs from a Wiskott-Aldrich Syndrome Patient Normalizes the Lymphoid Developmental and Functional Defects

Gene Correction of iPSCs from a Wiskott-Aldrich Syndrome Patient Normalizes the Lymphoid Developmental and Functional Defects

Wiskott-Aldrich syndrome (WAS) is an X-linked primary immunodeficiency disease caused by mutations in the gene encoding the WAS protein (WASp). Here, induced pluripotent stem cells (iPSCs) were derived from a WAS patient (WAS-iPSC) and the endogenous chromosomal WAS locus was targeted with a wtWAS-2...

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Bibliographic Details
Main Authors: Tamara J. Laskowski, Yasmine Van Caeneghem, Rasoul Pourebrahim, Chao Ma, Zhenya Ni, Zita Garate, Ana M. Crane, Xuan Shirley Li, Wei Liao, Manuel Gonzalez-Garay, Jose Carlos Segovia, David E. Paschon, Edward J. Rebar, Michael C. Holmes, Dan Kaufman, Bart Vandekerckhove, Brian R. Davis
Format: Article
Language:English
Published: Elsevier 2016-08-01
Series:Stem Cell Reports
Subjects:
induced pluripotent stem cells
genome editing
immune deficiency
T cells
Online Access:http://www.sciencedirect.com/science/article/pii/S2213671116300959

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