BCSH/BSBMT guideline: diagnosis and management of veno‐occlusive disease (sinusoidal obstruction syndrome) following haematopoietic stem cell transplantation von Dignan, Fiona L., Wynn, Robert F., Hadzic, Nedim, Karani, John, Quaglia, Alberto, Pagliuca, Antonio, Veys, Paul, Potter, Michael N.

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CTP synthase 1 deficiency in humans reveals its central role in lymphocyte proliferation von Martin, Emmanuel, Palmic, Noé, Sanquer, Sylvia, Lenoir, Christelle, Hauck, Fabian, Mongellaz, Cédric, Fabrega, Sylvie, Nitschké, Patrick, Esposti, Mauro Degli, Schwartzentruber, Jeremy, Taylor, Naomi, Majewski, Jacek, Jabado, Nada, Wynn, Robert F., Picard, Capucine, Fischer, Alain, Arkwright, Peter D., Latour, Sylvain

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Hematopoietic stem cell transplantation for CTLA4 deficiency von Slatter, Mary A., MD, Engelhardt, Karin R., PhD, Burroughs, Lauri M., MD, Arkwright, Peter D., DPhil, Nademi, Zohreh, PhD, Skoda-Smith, Suzanne, MD, Hagin, David, PhD, Kennedy, Alan, PhD, Barge, Dawn, PhD, Flood, Terence, MD, Abinun, Mario, PhD, Wynn, Robert F., MD, Gennery, Andrew R., MD, Cant, Andrew J., MD, Sansom, David, PhD, Hambleton, Sophie, DPhil, Torgerson, Troy R., MD

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Hematopoietic Stem Cell Transplantation in Inborn Errors of Metabolism von Tan, Emily Y., Boelens, Jaap Jan, Jones, Simon A., Wynn, Robert F.

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Study of Telomere Length Reveals Rapid Aging of Human Marrow Stromal Cells following In Vitro Expansion von Baxter, Melissa A., Wynn, Robert F., Jowitt, Simon N., Wraith, J. Ed, Fairbairn, Leslie J., Bellantuono, Ilaria

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Enzyme replacement therapy and hematopoietic stem cell transplant: a new paradigm of treatment in Wolman disease von Potter, Jane E, Petts, Gemma, Ghosh, Arunabha, White, Fiona J, Kinsella, Jane L, Hughes, Stephen, Roberts, Jane, Hodgkinson, Adam, Brammeier, Kathryn, Church, Heather, Merrigan, Christine, Hughes, Joanne, Evans, Pamela, Campbell, Helen, Bonney, Denise, Newman, William G, Bigger, Brian W, Broomfield, Alexander, Jones, Simon A, Wynn, Robert F

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Myeloid/Microglial Driven Autologous Hematopoietic Stem Cell Gene Therapy Corrects a Neuronopathic Lysosomal Disease von Sergijenko, Ana, Langford-Smith, Alexander, Liao, Ai Y, Pickford, Claire E, McDermott, John, Nowinski, Gabriel, Langford-Smith, Kia J, Merry, Catherine LR, Jones, Simon A, Wraith, J Edmond, Wynn, Robert F, Wilkinson, Fiona L, Bigger, Brian W

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A small proportion of mesenchymal stem cells strongly expresses functionally active CXCR4 receptor capable of promoting migration to bone marrow von Wynn, Robert F., Hart, Claire A., Corradi-Perini, Carla, O'Neill, Liam, Evans, Caroline A., Wraith, J. Ed, Fairbairn, Leslie J., Bellantuono, Ilaria

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Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure von de Ru, Minke H, Boelens, Jaap J, Das, Anibh M, Jones, Simon A, van der Lee, Johanna H, Mahlaoui, Nizar, Mengel, Eugen, Offringa, Martin, O'Meara, Anne, Parini, Rossella, Rovelli, Attilio, Sykora, Karl-Walter, Valayannopoulos, Vassili, Vellodi, Ashok, Wynn, Robert F, Wijburg, Frits A

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A novel syndrome of congenital sideroblastic anemia, B-cell immunodeficiency, periodic fevers, and developmental delay (SIFD) von Wiseman, Daniel H., May, Alison, Jolles, Stephen, Connor, Philip, Powell, Colin, Heeney, Matthew M., Giardina, Patricia J., Klaassen, Robert J., Chakraborty, Pranesh, Geraghty, Michael T., Major-Cook, Nathalie, Kannengiesser, Caroline, Thuret, Isabelle, Thompson, Alexis A., Marques, Laura, Hughes, Stephen, Bonney, Denise K., Bottomley, Sylvia S., Fleming, Mark D., Wynn, Robert F.

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Successful cure of C1q deficiency in human subjects treated with hematopoietic stem cell transplantation von Arkwright, Peter D., MD, PhD, Riley, Philip, MD, Hughes, Stephen M., MD, PhD, Alachkar, Hana, MD, Wynn, Robert F., MD

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Hematopoietic stem cell and gene therapy corrects primary neuropathology and behavior in mucopolysaccharidosis IIIA mice von Langford-Smith, Alexander, Wilkinson, Fiona L, Langford-Smith, Kia J, Holley, Rebecca J, Sergijenko, Ana, Howe, Steven J, Bennett, William R, Jones, Simon A, Wraith, Je, Merry, Catherine Lr, Wynn, Robert F, Bigger, Brian W

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Improved Metabolic Correction in Patients with Lysosomal Storage Disease Treated with Hematopoietic Stem Cell Transplant Compared with Enzyme Replacement Therapy von Wynn, Robert F., MD, Wraith, J. Ed, FRCPCH, Mercer, Jean, O'Meara, Anne, FRCPI, Tylee, Karen, BSc, Thornley, Margaret, Church, Heather J., PhD, Bigger, Brian W., PhD

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Use of Enzyme Replacement Therapy (Laronidase) before Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis I: Experience in 18 Patients von Wynn, Robert F., MD, Mercer, Jean, RGN, Page, Joanne, BSc, Carr, Trevor F., PhD, Jones, Simon, MRCPCH, Wraith, J. Edmond, FRCPCH

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Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler’s syndrome after pharmacological enzyme replacement therapy von AMEER SAIF, Muhammad, BIGGER, Brian W, BROOKES, Karen E, MERCER, Jean, TYLEE, Karen L, CHURCH, Heather J, BONNEY, Denise K, JONES, Simon, ED WRAITH, J, WYNN, Robert F

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Female mucopolysaccharidosis IIIA mice exhibit hyperactivity and a reduced sense of danger in the open field test von Langford-Smith, Alex, Langford-Smith, Kia J, Jones, Simon A, Wynn, Robert F, Wraith, J E, Wilkinson, Fiona L, Bigger, Brian W

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Signal one and two blockade are both critical for non-myeloablative murine HSCT across a major histocompatibility complex barrier von Langford-Smith, Kia J, Sandiford, Zara, Langford-Smith, Alex, Wilkinson, Fiona L, Jones, Simon A, Wraith, J Ed, Wynn, Robert F, Bigger, Brian W

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Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study von Aldenhoven, Mieke, Wynn, Robert F., Orchard, Paul J., O’Meara, Anne, Veys, Paul, Fischer, Alain, Valayannopoulos, Vassili, Neven, Benedicte, Rovelli, Attilio, Prasad, Vinod K., Tolar, Jakub, Allewelt, Heather, Jones, Simon A., Parini, Rossella, Renard, Marleen, Bordon, Victoria, Wulffraat, Nico M., de Koning, Tom J., Shapiro, Elsa G., Kurtzberg, Joanne, Boelens, Jaap Jan

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Haematopoietic stem cell transplantation in inborn errors of metabolism von Chiesa, Robert, Wynn, Robert F, Veys, Paul

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Transplantation in inborn errors of metabolism: current considerations and future perspectives von Boelens, Jaap Jan, Orchard, Paul J., Wynn, Robert F.

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