Research ArticleGene Therapy

Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency

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Science Translational Medicine  20 Apr 2016:
Vol. 8, Issue 335, pp. 335ra57
DOI: 10.1126/scitranslmed.aad8856

Article Information

vol. 8 no. 335 335ra57

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History: 
  • Received for publication November 16, 2015
  • Accepted for publication March 3, 2016

Author Information

  1. Suk See De Ravin1,*,
  2. Xiaolin Wu2,
  3. Susan Moir3,
  4. Lela Kardava3,
  5. Sandra Anaya-O’Brien1,
  6. Nana Kwatemaa1,
  7. Patricia Littel1,
  8. Narda Theobald1,
  9. Uimook Choi1,
  10. Ling Su2,
  11. Martha Marquesen1,
  12. Dianne Hilligoss1,
  13. Janet Lee1,
  14. Clarissa M. Buckner3,
  15. Kol A. Zarember1,
  16. Geraldine O’Connor4,
  17. Daniel McVicar4,
  18. Douglas Kuhns2,
  19. Robert E. Throm5,
  20. Sheng Zhou5,
  21. Luigi D. Notarangelo6,
  22. I. Celine Hanson7,
  23. Mort J. Cowan8,
  24. Elizabeth Kang1,
  25. Coleen Hadigan3,
  26. Michael Meagher5,
  27. John T. Gray9,
  28. Brian P. Sorrentino5, and
  29. Harry L. Malech1,*,
  1. 1Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
  2. 2Cancer Research Technology Program, Leidos Biomedical Research Inc., Frederick National Laboratory for Cancer Research, Frederick, MD 21702, USA.
  3. 3Laboratory of Immunoregulation, NIAID, NIH, Bethesda, MD 20892, USA.
  4. 4Cancer and Inflammation Program, National Cancer Institute Frederick, Frederick, MD 21702, USA.
  5. 5Department of Hematology, St. Jude Children’s Research Hospital, Memphis, TN 38105, USA.
  6. 6Division of Immunology, Boston Children’s Hospital, Harvard Medical School, Boston, MA 02115, USA.
  7. 7Texas Children’s Hospital, Houston, TX 77030, USA.
  8. 8Department of Pediatrics, Benioff Children’s Hospital, and University of California, San Francisco, San Francisco, CA, USA.
  9. 9Audentes Therapeutics, San Francisco, CA 94101, USA.
  1. *Corresponding author. E-mail: sderavin{at}niaid.nih.gov (S.S.D.R.); hmalech{at}niaid.nih.gov (H.L.M.)
    • These authors share co-senior authorship.

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