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

Alessandra Biffi

Research output: Contribution to journalArticle

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.

Original languageEnglish
Pages (from-to)348-349
Number of pages2
JournalCell Stem Cell
Volume16
Issue number4
DOIs
Publication statusPublished - Apr 2 2015

ASJC Scopus subject areas

  • Cell Biology
  • Molecular Medicine
  • Genetics
  • Medicine(all)

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