14800-DvRappard

23 General introduction 1 33. Kingsley PB, Shah TC, Woldenberg R. Identification of diffuse and focal brain lesions by clinical magnetic resonance spectroscopy. NMR Biomed . 2006;19:435-462. 34. van der Voorn JP, Pouwels PJ, Hart AA, Serrarens J, Willemsen MA, Kremer HP, et al. Childhood whitematter disorders: quantitativeMR imaging and spectroscopy. Radiology. 2006;241:510- 517. 35. Kruse B, Hanefeld F, Christen HJ, Bruhn H, Michaelis T, Hanicke W, et al. Alterations of brain metabolites in metachromatic leukodystrophy as detected by localized proton magnetic resonance spectroscopy in vivo. J Neurol. 1993;241:68-74. 36. Beaulieu C. The basis of anisotropic water diffusion in the nervous system - a technical review. NMR Biomed . 2002;15:435-455. 37. Stroobants S, Gerlach D, Matthes F, Hartmann D, Fogh J, Gieselmann V, et al. Intracerebroventricular enzyme infusion corrects central nervous system pathology and dysfunction in a mouse model of metachromatic leukodystrophy. Hum Mol Genet. 2011;20:2760-2769. 38. Aldenhoven M, Wynn RF, Orchard PJ, O'Meara A, Veys P, Fischer A, et al. Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study . Blood . 2015;125:2164-2172. 39. MartinHR,PoeMD,ProvenzaleJM,KurtzbergJ,MendizabalA,EscolarML.Neurodevelopmental outcomes of umbilical cord blood transplantation in metachromatic leukodystrophy. Biol Blood Marrow Transplant . 2013;19:616-624. 40. Biffi A, Montini E, Lorioli L, Cesani M, Fumagalli F, Plati T, et al. Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science. 2013;341:1233158.

RkJQdWJsaXNoZXIy MTk4NDMw