CRISPR-based gene editing enables FOXP3 gene repair in IPEX patient cells

  • M. Goodwin
  • , E. Lee
  • , U. Lakshmanan
  • , S. Shipp
  • , L. Froessl
  • , F. Barzaghi
  • , L. Passerini
  • , M. Narula
  • , A. Sheikali
  • , C. M. Lee
  • , G. Bao
  • , C. S. Bauer
  • , H. K. Miller
  • , M. Garcia-Lloret
  • , M. J. Butte
  • , A. Bertaina
  • , A. Shah
  • , M. Pavel-Dinu
  • , A. Hendel
  • , M. Porteus
  • M. G. Roncarolo, R. Bacchetta

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Biochemistry, Genetics and Molecular Biology