Clinical translation of TALENS: Treating SCID-X1 by gene editing in iPSCs
- PMID: 25842973
- DOI: 10.1016/j.stem.2015.03.009
Clinical translation of TALENS: Treating SCID-X1 by gene editing in iPSCs
Abstract
Mutations causing X-linked severe combined immunodeficiency (SCID-X1) reduce immune cell populations and function and may be amenable to targeted gene correction strategies. Now in Cell Stem Cell, Menon et al. (2015) correct SCID-X1-related blood differentiation defects by TALEN-mediated genome editing in patient-derived iPSCs, suggesting a possible strategy for autologous cell therapy of SCID-X1.
Copyright © 2015 Elsevier Inc. All rights reserved.
Comment on
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Lymphoid regeneration from gene-corrected SCID-X1 subject-derived iPSCs.Cell Stem Cell. 2015 Apr 2;16(4):367-72. doi: 10.1016/j.stem.2015.02.005. Epub 2015 Mar 12. Cell Stem Cell. 2015. PMID: 25772073 Free PMC article.
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