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Molecular Therapy : the Journal of the American Society of Gene Therapy|May 2, 2012
Hematopoietic stem cell and gene therapy corrects primary neuropathology and behavior in mucopolysaccharidosis IIIA miceAlexander Langford-Smith, Fiona L Wilkinson, Kia J Langford-Smith, et al.
Bone Marrow Transplantation|May 29, 2007
Outcomes of hematopoietic stem cell transplantation for Hurler's syndrome in Europe: a risk factor analysis for graft failureJ J Boelens, R F Wynn, A O'Meara, et al.
Plos One|May 5, 2012
Neuropathology in mouse models of mucopolysaccharidosis type I, IIIA and IIIBFiona L Wilkinson, Rebecca J Holley, Kia J Langford-Smith, et al.
Bone Marrow Transplantation|April 7, 2005
Survey on haematopoietic stem cell transplantation for children in EuropeM Miano, R Cancedda, O Hartmann, et al.
Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation|February 25, 2015
Hematopoietic cell transplantation for mucopolysaccharidosis patients is safe and effective: results after implementation of international guidelinesMieke Aldenhoven, Simon A Jones, Denise Bonney, et al.
Blood Cell Therapy|October 27, 2023
A decade of excellent transplant survival in children with inherited metabolic diseases: A report from a single metabolic transplant centre in EuropeSu Han Lum, Andrew Will, Heather J Church, et al.
Blood Advances|January 4, 2018
Quality of life of Hurler syndrome patients after successful hematopoietic stem cell transplantationMieke Aldenhoven, Brigitte T A van den Broek, Robert F Wynn, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|June 11, 2013
Myeloid/Microglial driven autologous hematopoietic stem cell gene therapy corrects a neuronopathic lysosomal diseaseAna Sergijenko, Alexander Langford-Smith, Ai Y Liao, et al.
Familial Cancer|July 26, 2012
Heterozygote FANCD2 mutations associated with childhood T Cell ALL and testicular seminomaStephanie Smetsers, Joanne Muter, Claire Bristow, et al.
Nature|May 30, 2014
CTP synthase 1 deficiency in humans reveals its central role in lymphocyte proliferationEmmanuel Martin, Noé Palmic, Sylvia Sanquer, et al.
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