Research ArticleGene Therapy

Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1

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Science Translational Medicine  11 Oct 2017:
Vol. 9, Issue 411, eaan0820
DOI: 10.1126/scitranslmed.aan0820

Article Information

vol. 9 no. 411

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History: 
  • Received for publication March 1, 2017
  • Resubmitted June 26, 2017
  • Accepted for publication September 12, 2017

Author Information

  1. Giulia Schiroli1,2,
  2. Samuele Ferrari1,2,
  3. Anthony Conway3,
  4. Aurelien Jacob1,
  5. Valentina Capo1,
  6. Luisa Albano1,
  7. Tiziana Plati1,
  8. Maria C. Castiello1,
  9. Francesca Sanvito4,
  10. Andrew R. Gennery5,
  11. Chiara Bovolenta6,
  12. Rahul Palchaudhuri7,8,
  13. David T. Scadden8,
  14. Michael C. Holmes3,
  15. Anna Villa1,9,
  16. Giovanni Sitia10,
  17. Angelo Lombardo1,2,
  18. Pietro Genovese1,*, and
  19. Luigi Naldini1,2,*,
  1. 1San Raffaele Telethon Institute for Gene Therapy, 20132 Milan, Italy.
  2. 2Vita-Salute San Raffaele University, 20132 Milan, Italy.
  3. 3Sangamo Therapeutics, Richmond, CA 94804, USA.
  4. 4Pathology Unit, Department of Oncology, San Raffaele Scientific Institute, 20132 Milan, Italy.
  5. 5Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne NE2 4HH, UK.
  6. 6MolMed S.p.A., 20132 Milan, Italy.
  7. 7Magenta Therapeutics, Cambridge, MA 02139, USA.
  8. 8Harvard Stem Cell Institute, Cambridge, MA 02138, USA.
  9. 9National Research Council, Institute of Genetic and Biomedical Research Milan Unit, 20138 Milan, Italy.
  10. 10San Raffaele Scientific Institute, 20132 Milan, Italy.
  1. Corresponding author. Email: genovese.pietro{at}hsr.it (P.G.); naldini.luigi{at}hsr.it (L.N.)
    • * These authors contributed equally to this work.

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    Nov 201761955153
    Dec 20172092352