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Comment
. 2015 Apr 2;16(4):348-9.
doi: 10.1016/j.stem.2015.03.009.

Clinical translation of TALENS: Treating SCID-X1 by gene editing in iPSCs

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Clinical translation of TALENS: Treating SCID-X1 by gene editing in iPSCs

Alessandra Biffi. Cell Stem Cell. .
Free article

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.

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  • Lymphoid regeneration from gene-corrected SCID-X1 subject-derived iPSCs.
    Menon T, Firth AL, Scripture-Adams DD, Galic Z, Qualls SJ, Gilmore WB, Ke E, Singer O, Anderson LS, Bornzin AR, Alexander IE, Zack JA, Verma IM. Menon T, et al. 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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