Research ArticleGene Therapy

CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease

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Science Translational Medicine  11 Jan 2017:
Vol. 9, Issue 372, eaah3480
DOI: 10.1126/scitranslmed.aah3480

Article Information

vol. 9 no. 372

PubMed: 
Print ISSN: 
Online ISSN: 
History: 
  • Received for publication June 13, 2016
  • Resubmitted September 9, 2016
  • Accepted for publication August 17, 2016

Author Information

  1. Suk See De Ravin1,*,,
  2. Linhong Li2,*,
  3. Xiaolin Wu3,
  4. Uimook Choi1,
  5. Cornell Allen2,
  6. Sherry Koontz1,
  7. Janet Lee1,
  8. Narda Theobald-Whiting1,
  9. Jessica Chu1,
  10. Mary Garofalo1,
  11. Colin Sweeney1,
  12. Lela Kardava4,
  13. Susan Moir4,
  14. Angelia Viley2,
  15. Pachai Natarajan2,
  16. Ling Su3,
  17. Douglas Kuhns1,
  18. Kol A. Zarember1,
  19. Madhusudan V. Peshwa2 and
  20. Harry L. Malech1,
  1. 1Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD 20892, USA.
  2. 2MaxCyte Inc., Gaithersburg, MD 20878, USA.
  3. 3Cancer Research Technology Program, Leidos Biomedical Research Inc., Frederick, MD 21701, USA.
  4. 4Laboratory of Immunoregulation, National Institutes of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD 20892, USA.
  1. Corresponding author. Email: sderavin{at}niaid.nih.gov (S.S.D.R.); hmalech{at}nih.gov (H.L.M.)
    • * These authors contributed equally to this work.

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