Research Article

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

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Science  23 Aug 2013:
Vol. 341, Issue 6148, 1233158
DOI: 10.1126/science.1233158

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vol. 341 no. 6148

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History: 
  • Received for publication November 26, 2012
  • Accepted for publication June 25, 2013

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  1. Alessandra Biffi1,2,3,*,,
  2. Eugenio Montini1,*,
  3. Laura Lorioli1,2,3,4,
  4. Martina Cesani1,
  5. Francesca Fumagalli2,4,5,
  6. Tiziana Plati1,
  7. Cristina Baldoli6,
  8. Sabata Martino7,
  9. Andrea Calabria1,
  10. Sabrina Canale2,
  11. Fabrizio Benedicenti1,
  12. Giuliana Vallanti8,
  13. Luca Biasco1,
  14. Simone Leo9,
  15. Nabil Kabbara10,
  16. Gianluigi Zanetti9,
  17. William B. Rizzo11,
  18. Nalini A. L. Mehta12,
  19. Maria Pia Cicalese2,3,
  20. Miriam Casiraghi2,
  21. Jaap J. Boelens13,
  22. Ubaldo Del Carro5,
  23. David J. Dow12,
  24. Manfred Schmidt14,
  25. Andrea Assanelli3,15,
  26. Victor Neduva12,
  27. Clelia Di Serio4,
  28. Elia Stupka16,
  29. Jason Gardner17,
  30. Christof von Kalle14,
  31. Claudio Bordignon4,8,
  32. Fabio Ciceri3,15,
  33. Attilio Rovelli18,
  34. Maria Grazia Roncarolo1,2,3,4,
  35. Alessandro Aiuti1,2,3,19,
  36. Maria Sessa2,5,
  37. Luigi Naldini1,4,
  1. 1San Raffaele Telethon Institute for Gene Therapy (TIGET), San Raffaele Scientific Institute, 20132 Milan, Italy.
  2. 2TIGET Pediatric Clinical Research Unit, Division of Regenerative Medicine, Stem Cells and Gene Therapy, San Raffaele Scientific Institute, 20132 Milan, Italy.
  3. 3Pediatric Immunohematology and Bone Marrow Transplant Unit, San Raffaele Scientific Institute, 20132 Milan, Italy.
  4. 4Vita-Salute San Raffaele University, 20132 Milan, Italy.
  5. 5Neurology Unit, Department of Neurology, San Raffaele Scientific Institute, 20132 Milan, Italy.
  6. 6Neuroradiology Unit, Head and Neck Department, San Raffaele Scientific Institute, 20132 Milan, Italy.
  7. 7Department of Experimental Medicine and Biochemical Sciences, University of Perugia, 06122 Perugia, Italy.
  8. 8MolMed, 20132 Milan, Italy.
  9. 9Distributed Computing Group, Center for Advanced Studies, Research and Development in Sardinia (CRS4), 09010 Pula, Italy.
  10. 10Pediatric Hematology Oncology Division, Rafic Hariri University Hospital, Beirut, Lebanon.
  11. 11Department of Pediatrics, University of Nebraska Medical Center, Omaha, NE 68198, USA.
  12. 12Molecular and Cellular Technologies, GlaxoSmithKline, Stevenage 5G1 2NY, UK.
  13. 13Pediatric Blood and Marrow Transplantation Program, University Medical Center 3584 CX Utrecht, Netherlands.
  14. 14Department of Translational Oncology, National Center for Tumor Diseases and German Cancer Research Center, 69120 Heidelberg, Germany.
  15. 15Hematology and Bone Marrow Transplant Unit, San Raffaele Scientific Institute, 20132 Milan, Italy.
  16. 16Center for Translational Genomics and BioInformatics, San Raffaele Scientific Institute, 20132 Milan, Italy.
  17. 17Regenerative Medicine Discovery Performance Unit, GlaxoSmithKline Research and Development, King of Prussia, PA 19406, USA.
  18. 18Bone Marrow Transplant Unit, MBBM Foundation, Pediatric Department, Milano-Bicocca University at San Gerardo Hospital, 20052 Monza, Italy.
  19. 19University of Rome Tor Vergata, 00133 Rome, Italy.
  1. * These authors contributed equally to this work.

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