Research ArticleIMMUNOLOGY

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

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Science Advances  06 May 2020:
Vol. 6, no. 19, eaaz0571
DOI: 10.1126/sciadv.aaz0571

Article Information

vol. 6 no. 19

Online ISSN: 
History: 
  • Received for publication August 8, 2019
  • Accepted for publication February 20, 2020
  • .

Author Information

  1. M. Goodwin1,*,
  2. E. Lee1,2,*,
  3. U. Lakshmanan1,
  4. S. Shipp1,
  5. L. Froessl1,
  6. F. Barzaghi3,
  7. L. Passerini3,
  8. M. Narula1,
  9. A. Sheikali1,
  10. C. M. Lee4,
  11. G. Bao5,
  12. C. S. Bauer6,
  13. H. K. Miller6,
  14. M. Garcia-Lloret7,
  15. M. J. Butte7,
  16. A. Bertaina1,
  17. A. Shah1,
  18. M. Pavel-Dinu1,
  19. A. Hendel1,8,
  20. M. Porteus1,2,
  21. M. G. Roncarolo1,2 and
  22. R. Bacchetta1,
  1. 1Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.
  2. 2Institute for Stem Cell Biology and Regenerative Medicine (ISCBRM), Stanford University School of Medicine, Stanford, CA, USA.
  3. 3Division of Regenerative Medicine, Stem Cells and Gene Therapy, San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan, Italy.
  4. 4APC Microbiome Ireland, University College Cork, Cork, Ireland.
  5. 5Department of Bioengineering, George R. Brown School of Engineering, Rice University, Houston, TX, USA.
  6. 6Phoenix Children's Hospital, Phoenix, AZ, USA.
  7. 7Division of Immunology, Allergy, and Rheumatology, Department of Pediatrics, University of California, Los Angeles, Los Angeles, CA, USA.
  8. 8The Institute for Advanced Materials and Nanotechnology, The Mina and Everard Goodman Faculty of Life Sciences, Ramat-Gan 52900, Israel.
  1. Corresponding author. Email: rosab{at}stanford.edu
  • * These authors contributed equally to this work.

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