Chabannon et al Sci Transl Med 10 eaap9630 2018 11 April 2018 S C I E N C E T R

Chabannon et al sci transl med 10 eaap9630 2018 11

This preview shows page 8 - 9 out of 11 pages.

Chabannon et al., Sci. Transl. Med.10, eaap9630 (2018) 11 April 2018S C I E N C E T R A N S L A T I O N A L M E D I C I N E |R E V I E W8 of 10stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science326, 818–823 (2009).17. M. Cavazzana-Calvo, E. Payen, O. Negre, G. Wang, K. Hehir, F. Fusil, J. Down, M. Denaro, T. Brady, K. Westerman, R. Cavallesco, B. Gillet-Legrand, L. Caccavelli, R. Sgarra, L. Maouche-Chrétien, F. Bernaudin, R. Girot, R. Dorazio, G.-J. Mulder, A. Polack, A. Bank, J. Soulier, J. Larghero, N. Kabbara, B. Dalle, B. Gourmel, G. Socie, S. Chrétien, N. Cartier, P. Aubourg, A. Fischer, K. Cornetta, F. Galacteros, Y. Beuzard, E. Gluckman, F. Bushman, S. Hacein-Bey-Abina, P. Leboulch, Transfusion independence and HMGA2activation after gene therapy of human -thalassaemia. Nature467, 318–322 (2010).18. J.-A. Ribeil, S. Hacein-Bey-Abina, E. Payen, A. Magnani, M. Semeraro, E. Magrin, L. Caccavelli, B. Neven, P. Bourget, W. El Nemer, P. Bartolucci, L. Weber, H. Puy, J.-F. Meritet, D. Grevent, Y. Beuzard, S. Chrétien, T. Lefebvre, R. W. Ross, O. Negre, G. Veres, L. Sandler, S. Soni, M. de Montalembert, S. Blanche, P. Leboulch, M. Cavazzana, Gene therapy in a patient with sickle cell disease. N. Engl. J. Med.376, 848–855 (2017).19. E. A. Roselli, R. Mezzadra, M. C. Frittoli, G. Maruggi, E. Biral, F. Mavilio, F. Mastropietro, A. Amato, G. Tonon, C. Refaldi, M. D. Cappellini, M. Andreani, G. Lucarelli, M. G. Roncarolo, S. Marktel, G. Ferrari, Correction of -thalassemia major by gene transfer in haematopoietic progenitors of pediatric patients. EMBO Mol. Med.2, 315–328 (2010).20. L. Naldini, D. Trono, I. M. Verma, Lentiviral vectors, two decades later. Science353, 1101–1102 (2016).21. J. E. DiCarlo, A. Deeconda, S. H. Tsang, Viral vectors, engineered cells and the CRISPR revolution. Adv. Exp. Med. Biol.1016, 3–27 (2017).22. T. T. Smith, S. B. Stephan, H. F. Moffett, L. E. McKnight, W. Ji, D. Reiman, E. Bonagofski, M. E. Wohlfahrt, S. P. S. Pillai, M. T. Stephan, In situ programming of leukaemia-specific T cells using synthetic DNA nanocarriers. Nat. Nanotechnol.12, 813–820 (2017).23. J. Tipanee, Y. C. Chai, T. VandenDriessche, M. K. Chuah, Preclinical and clinical advances in transposon-based gene therapy. Biosci. Rep.37, BSR20160614 (2017).24. K. L. Gandy, Tolerance induction for solid organ grafts with donor-derived hematopoietic reconstitution. Immunol. Res.22, 147–164 (2000).25. T. Kawai, D. H. Sachs, M. Sykes, A. B. Cosimi; Immune Tolerance Network, HLA-mismatched renal transplantation without maintenance immunosuppression. N. Engl. J. Med.368, 1850–1852 (2013).26. R. A. Nash, G. J. Hutton, M. K. Racke, U. Popat, S. M. Devine, K. C. Steinmiller, L. M. Griffith, P. A. Muraro, H. Openshaw, P. H. Sayre, O. Stuve, D. L. Arnold, M. H. Wener, G. E. Georges, A. Wundes, G. H. Kraft, J. D. Bowen, High-dose immunosuppressive therapy and autologous HCT for relapsing-remitting MS. Neurology88, 842–852 (2017).27. M. P. Sormani, P. A. Muraro, I. Schiavetti, A. Signori, A. Laroni, R. Saccardi, G. L. Mancardi, Autologous hematopoietic stem cell transplantation in multiple sclerosis: A meta-analysis. Neurology88, 2115–2122 (2017).
Background image

Want to read all 11 pages?

Image of page 9

Want to read all 11 pages?

You've reached the end of your free preview.

Want to read all 11 pages?

  • Fall '13
  • The Land, Bone marrow, Hematopoietic stem cell transplantation, Science Translational Medicine, HSCT

  • Left Quote Icon

    Student Picture

  • Left Quote Icon

    Student Picture

  • Left Quote Icon

    Student Picture