Gene Correction of iPSCs from a Wiskott-Aldrich Syndrome Patient Normalizes the Lymphoid Developmental and Functional Defects
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- 作者：Tamara J. Laskowski1 ; 8 ; Yasmine Van Caeneghem2 ; 8 ; Rasoul Pourebrahim1 ; Chao Ma3 ; Zhenya Ni3 ; Zita Garate1 ; 4 ; 5 ; Ana M. Crane1 ; Xuan Shirley Li1 ; Wei Liao1 ; Manuel Gonzalez-Garay6 ; Jose Carlos Segovia4 ; 5 ; David E. Paschon7 ; Edward J. Rebar7 ; Michael C. Holmes7 ; Dan Kaufman3 ; 9 ; Bart Vandekerckhove2 ; Brian R. Davis1 ; brian.r.davis@uth.tmc.edu" class="auth_mail" title="E-mail the corresponding author
- 关键词：induced pluripotent stem cells ; genome editing ; immune deficiency ; T cells
- 刊名：Stem Cell Reports
- 出版年：2016
- 出版时间：9 August 2016
- 年：2016
- 卷：7
- 期：2
- 页码：139-148
- 全文大小：1521 K
文摘
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Targeted endogenous knockin of Wiskott-Aldrich syndrome (WAS) transgene in WAS-iPSCs
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Mutant WAS-iPSCs exhibited defective NK- and T-lymphoid cell development and function
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Correction of WAS-iPSCs restored lymphoid cell development and function
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These results suggest the potential therapeutic use of gene-corrected WAS-iPSCs