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FontName: OENMNO+AdvOT1ef757c0+20 Flags: Nonsymbolic FontBBox: -135, -187, 1010, 833 FontFile3: true Encoding: WinAnsiEncoding Font: BaseFont: OENMNL+AdvHelN-L FontSubset: true FirstChar: 38 LastChar: 223 FontDescriptor: FontName: OENMNL+AdvHelN-L Flags: Nonsymbolic FontBBox: -156, -208, 1041, 802 FontFile3: true Encoding: MacRomanEncoding XMP: application/pdf doi:10.1016/j.omtm.2018.01.001 Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE Rana Yadak Raquel Cabrera-Pérez Javier Torres-Torronteras Marianna Bugiani Joost C. Haeck Marshall W. Huston Elly Bogaerts Steffi Goffart Edwin H. Jacobs Merel Stok Lorena Leonardelli Luca Biasco Robert M. Verdijk Monique R. Bernsen George Ruijter Ramon Martí Gerard Wagemaker Niek P. van Til Irenaeus F.M. de Coo MNGIE thymidine phosphorylase hematopoietic stem cells lentiviral vectors gene therapy Molecular Therapy: Methods & Clinical Development, 8 (2018) 152-165. doi:10.1016/j.omtm.2018.01.001 Elsevier Ltd. journal Molecular Therapy: Methods & Clinical Development © 2018 The Authors. 2329-0501 8 16 March 2018 2018-03-16 152-165 152 165 10.1016/j.omtm.2018.01.001 https://doi.org/10.1016/j.omtm.2018.01.001 2010-04-23 true 10.1016/j.omtm.2018.01.001 elsevier.com sciencedirect.com VoR 6.5 10.1016/j.omtm.2018.01.001 noindex 2010-04-23 true sciencedirect.com elsevier.com Elsevier 2018-03-10T05:25:20+05:30 2018-03-10T05:26:47+05:30 2018-03-10T05:26:47+05:30 True Acrobat Distiller 8.1.0 (Windows) uuid:2eaafec5-4903-4827-96cd-0385e2d7e5ac uuid:cfb6055e-d51a-449d-bf6a-b82cce453dd7 http://creativecommons.org/licenses/by-nc-nd/4.0/ Pages: Page: Label: 152 Sequence: 1 Annotations: Annotation: Subtype: Link Rect: 104, 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