Lentiviral haematopoietic stem cell gene therapy (HSC-GT) for metachromatic leukodystrophy (MLD): Preliminary results from a clinical trial with a cryopreserved formulation of OTL-200

著录项

• 来源
  《Human gene therapy》 |2019年第11期|共2页
• 作者

  Calbi V.; Fumagalli F.; Acquati S.; Miglietta S.; Ciotti F.; Fraschini M.; Sarzana M.; Baldoli C.; Recupero S.; Zambon A.; Barzaghi F.; Ferrua F.; Cicalese M. P.; Migliavacca M.; Tucci F.; Gallo V.; La Marca G.; Silvani P.; Facchini M.; Locatelli S.; Antonioli G.; Zancan S.; Farinelli G.; Morena F.; Segovia J.; Schwab L. C.; Downey G.; Gabaldo M.; Martino S.; Ciceri F.; Sora M. G. Natali; Bernardo M. E.; Naldini L.; Aiuti A.;

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• 作者单位

  IRCCS San Raffaele Sci Inst San Raffaele Telethon Inst Gene Therapy SR TIGET Milan Italy;

  IRCCS San Raffaele Sci Inst San Raffaele Telethon Inst Gene Therapy SR TIGET Milan Italy;

  IRCCS San Raffaele Sci Inst San Raffaele Telethon Inst Gene Therapy SR TIGET Milan Italy;

  IRCCS San Raffaele Sci Inst San Raffaele Telethon Inst Gene Therapy SR TIGET Milan Italy;

  IRCCS San Raffaele Sci Inst Pediat Immunohematol Unit Milan Italy;

  IRCCS San Raffaele Sci Inst Pediat Immunohematol Unit Milan Italy;

  IRCCS San Raffaele Sci Inst Pediat Immunohematol Unit Milan Italy;

  IRCCS San Raffaele Sci Inst Neuroradiol Unit Milan Italy;

  IRCCS San Raffaele Sci Inst Pediat Immunohematol Unit Milan Italy;

  IRCCS San Raffaele Sci Inst Dept Neurol Milan Italy;

  IRCCS San Raffaele Sci Inst San Raffaele Telethon Inst Gene Therapy SR TIGET Milan Italy;

  IRCCS San Raffaele Sci Inst San Raffaele Telethon Inst Gene Therapy SR TIGET Milan Italy;

  IRCCS San Raffaele Sci Inst San Raffaele Telethon Inst Gene Therapy SR TIGET Milan Italy;

  IRCCS San Raffaele Sci Inst San Raffaele Telethon Inst Gene Therapy SR TIGET Milan Italy;

  IRCCS San Raffaele Sci Inst San Raffaele Telethon Inst Gene Therapy SR TIGET Milan Italy;

  IRCCS San Raffaele Sci Inst San Raffaele Telethon Inst Gene Therapy SR TIGET Milan Italy;

  Meyer Hosp Florence Lab Diag Cent Nervous Syst Dis &

  Metab Disorders Florence Italy;

  IRCCS San Raffaele Sci Inst Dept Anesthesia &

  Crit Care Milan Italy;

  IRCCS San Raffaele Sci Inst San Raffaele Telethon Inst Gene Therapy SR TIGET Milan Italy;

  IRCCS San Raffaele Sci Inst San Raffaele Telethon Inst Gene Therapy SR TIGET Milan Italy;

  IRCCS San Raffaele Sci Inst San Raffaele Telethon Inst Gene Therapy SR TIGET Milan Italy;

  IRCCS San Raffaele Sci Inst San Raffaele Telethon Inst Gene Therapy SR TIGET Milan Italy;

  IRCCS San Raffaele Sci Inst San Raffaele Telethon Inst Gene Therapy SR TIGET Milan Italy;

  Univ Perugia Dept Chem Biol &

  Biotechnol Perugia Italy;

  Orchard Therapeut Ltd London England;

  Orchard Therapeut Ltd London England;

  Orchard Therapeut Ltd London England;

  IRCCS San Raffaele Sci Inst San Raffaele Telethon Inst Gene Therapy SR TIGET Milan Italy;

  Univ Perugia Dept Chem Biol &

  Biotechnol Perugia Italy;

  IRCCS San Raffaele Sci Inst Unit Hematol &

  Bone Marrow Transplantat Milan Italy;

  IRCCS San Raffaele Sci Inst Dept Neurol Milan Italy;

  IRCCS San Raffaele Sci Inst San Raffaele Telethon Inst Gene Therapy SR TIGET Milan Italy;

  IRCCS San Raffaele Sci Inst San Raffaele Telethon Inst Gene Therapy SR TIGET Milan Italy;

  IRCCS San Raffaele Sci Inst San Raffaele Telethon Inst Gene Therapy SR TIGET Milan Italy;

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• 原文格式 PDF
• 正文语种 eng
• 中图分类 治疗学;
• 关键词