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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Detalles Bibliográficos
Autores principales: Laskowski, Tamara J., Van Caeneghem, Yasmine, Pourebrahim, Rasoul, Ma, Chao, Ni, Zhenya, Garate, Zita, Crane, Ana M., Li, Xuan Shirley, Liao, Wei, Gonzalez-Garay, Manuel, Segovia, Jose Carlos, Paschon, David E., Rebar, Edward J., Holmes, Michael C., Kaufman, Dan, Vandekerckhove, Bart, Davis, Brian R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2016
Materias:
Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4982969/
https://www.ncbi.nlm.nih.gov/pubmed/27396937
http://dx.doi.org/10.1016/j.stemcr.2016.06.003

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