Hematopoietic Stem Cell Transplantation in Inborn Errors of Metabolism

Por Tan, Emily Y., Boelens, Jaap Jan, Jones, Simon A., Wynn, Robert F.
Publicado no Front Pediatr (2019)

Female Mucopolysaccharidosis IIIA Mice Exhibit Hyperactivity and a Reduced Sense of Danger in the Open Field Test

Por Langford-Smith, Alex, Langford-Smith, Kia J., Jones, Simon A., Wynn, Robert F., Wraith, J. E., Wilkinson, Fiona L., Bigger, Brian W.
Publicado em 2011

Immune cytopenia post–cord transplant in Hurler syndrome is a forme fruste of graft rejection

Por Deambrosis, David, Lum, Su Han, Hum, Ryan M., Poulton, Kay, Ogden, Wendy, Jones, Simon, Stanworth, Simon, Bonney, Denise, Hiwarkar, Prashant, Wynn, Robert F.
Publicado no Blood Adv (2019)

Signal One and Two Blockade Are Both Critical for Non-Myeloablative Murine HSCT across a Major Histocompatibility Complex Barrier

Por Langford-Smith, Kia J., Sandiford, Zara, Langford-Smith, Alex, Wilkinson, Fiona L., Jones, Simon A., Wraith, J. Ed, Wynn, Robert F., Bigger, Brian W.
Publicado em 2013

Enzyme replacement therapy prior to haematopoietic stem cell transplantation in Mucopolysaccharidosis Type I: 10 year combined experience of 2 centres

Por Ghosh, Arunabha, Miller, Weston, Orchard, Paul J., Jones, Simon A., Mercer, Jean, Church, Heather J., Tylee, Karen, Lund, Troy, Bigger, Brian W., Tolar, Jakub, Wynn, Robert F.
Publicado no Mol Genet Metab (2016)

Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler’s syndrome after pharmacological enzyme replacement therapy

Por Saif, Muhammad Ameer, Bigger, Brian W., Brookes, Karen E, Mercer, Jean, Tylee, Karen L., Church, Heather J., Bonney, Denise K., Jones, Simon, Wraith, J. Ed, Wynn, Robert F.
Publicado em 2012

BCSH/BSBMT/UK clinical virology network guideline: diagnosis and management of common respiratory viral infections in patients undergoing treatment for haematological malignancies...

Por Dignan, Fiona L., Clark, Andrew, Aitken, Celia, Gilleece, Maria, Jayakar, Vishal, Krishnamurthy, Pramila, Pagliuca, Antonio, Potter, Michael N., Shaw, Bronwen, Skinner, Roderick, Turner, Andrew, Wynn, Robert F., Coyle, Peter
Publicado no Br J Haematol (2016)

Heparan Sulfate Inhibits Hematopoietic Stem and Progenitor Cell Migration and Engraftment in Mucopolysaccharidosis I

Por Watson, H. Angharad, Holley, Rebecca J., Langford-Smith, Kia J., Wilkinson, Fiona L., van Kuppevelt, Toin H., Wynn, Robert F., Wraith, J. Edmond, Merry, Catherine L. R., Bigger, Brian W.
Publicado no J Biol Chem (2014)

Central nervous system abnormalities in Fanconi anaemia: patterns and frequency on magnetic resonance imaging

Por Stivaros, Stavros M, Alston, Robert, Wright, Neville B, Chandler, Kate, Bonney, Denise, Wynn, Robert F, Will, Andrew M, Punekar, Maqsood, Loughran, Sean, Kilday, John-Paul, Schindler, Detlev, Patel, Leena, Meyer, Stefan
Publicado no Br J Radiol (2015)

Sleep disordered breathing in mucopolysaccharidosis I: a multivariate analysis of patient, therapeutic and metabolic correlators modifying long term clinical outcome

Por Pal, Abhijit Ricky, Langereis, Eveline J, Saif, Muhammad A, Mercer, Jean, Church, Heather J, Tylee, Karen L, Wynn, Robert F, Wijburg, Frits A, Jones, Simon A, Bruce, Iain A, Bigger, Brian W
Publicado no Orphanet J Rare Dis (2015)

Hematopoietic Stem Cell and Gene Therapy Corrects Primary Neuropathology and Behavior in Mucopolysaccharidosis IIIA Mice

Por 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
Publicado em 2012

Quality of life of Hurler syndrome patients after successful hematopoietic stem cell transplantation

Por Aldenhoven, Mieke, van den Broek, Brigitte T. A., Wynn, Robert F., O’Meara, Anne, Veys, Paul, Rovelli, Attilio, Jones, Simon A., Parini, Rossella, van Hasselt, Peter M., Renard, Marleen, Bordon, Victoria, de Koning, Tom J., Boelens, Jaap Jan
Publicado no Blood Adv (2017)

Myeloid/Microglial Driven Autologous Hematopoietic Stem Cell Gene Therapy Corrects a Neuronopathic Lysosomal Disease

Por 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
Publicado em 2013

The influence of stem cell source on transplant outcomes for pediatric patients with acute myeloid leukemia

Por Keating, Amy K., Langenhorst, Jurgen, Wagner, John E., Page, Kristin M., Veys, Paul, Wynn, Robert F., Stefanski, Heather, Elfeky, Reem, Giller, Roger, Mitchell, Richard, Milano, Filippo, O'Brien, Tracey A., Dahlberg, Ann, Delaney, Colleen, Kurtzberg, Joanne, Verneris, Michael R., Boelens, Jaap Jan
Publicado no Blood Adv (2019)

Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure

Por 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
Publicado em 2011

A novel syndrome of congenital sideroblastic anemia, B-cell immunodeficiency, periodic fevers, and developmental delay (SIFD)

Por 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.
Publicado em 2013

Impaired lymphocyte function and differentiation in CTPS1-deficient patients result from a hypomorphic homozygous mutation

Por Martin, Emmanuel, Minet, Norbert, Boschat, Anne-Claire, Sanquer, Sylvia, Sobrino, Steicy, Lenoir, Christelle, de Villartay, Jean Pierre, Leite-de-Moraes, Maria, Picard, Capucine, Soudais, Claire, Bourne, Tim, Hambleton, Sophie, Hughes, Stephen M., Wynn, Robert F., Briggs, Tracy A., Patel, Smita, Lawrence, Monica G., Fischer, Alain, Arkwright, Peter D., Latour, Sylvain
Publicado no JCI Insight (2020)

Impact of immune modulation with in vivo T-cell depletion and myleoablative total body irradiation conditioning on outcomes after unrelated donor transplantation for childhood acut...

Por Veys, Paul, Wynn, Robert F., Ahn, Kwang Woo, Samarasinghe, Sujith, He, Wensheng, Bonney, Denise, Craddock, John, Cornish, Jacqueline, Davies, Stella M., Dvorak, Christopher C., Duerst, Reggie E., Gross, Thomas G., Kapoor, Neena, Kitko, Carrie, Krance, Robert A., Leung, Wing, Lewis, Victor A., Steward, Colin, Wagner, John E., Carpenter, Paul A., Eapen, Mary
Publicado em 2012

Targeted busulfan-based reduced-intensity conditioning and HLA-matched HSCT cure hemophagocytic lymphohistiocytosis

Por Felber, Matthias, Steward, Colin G., Kentouche, Karim, Fasth, Anders, Wynn, Robert F., Zeilhofer, Ulrike, Haunerdinger, Veronika, Volkmer, Benjamin, Prader, Seraina, Gruhn, Bernd, Ehl, Stephan, Lehmberg, Kai, Müller, Daniel, Gennery, Andrew R., Albert, Michael H., Hauck, Fabian, Rao, Kanchan, Veys, Paul, Hassan, Moustapha, Lankester, Arjan C., Schmid, Jana Pachlopnik, Hauri-Hohl, Mathias M., Güngör, Tayfun
Publicado no Blood Adv (2020)

A New Harmonized Approach to Estimate Busulfan Exposure Predicts Survival and Toxicity after Hematopoietic Cell Transplantation in Children and Young Adults: a Multicenter Retrospe...

Por Bartelink, I.H., Lalmohamed, Arief, van Reij, Elisabeth M.L., Dvorak, Chris C., Savic, Rada M., Zwaveling, Juliette, Bredius, Robbert. G.M., Egberts, Antoine C.G., Bierings, M., Kletzel, M., Shaw, Peter J., Nath, Christa E., Hempel, George, Ansari, M., Krajinovic, M., Theoret, Yves, Duval, Michel, Keizer, Ron J., Bittencourt, Henriette, Hassan, Moustapha, Güngör, Tayfun, Wynn, Robert F., Veys, Paul, Cuvelier, Geoff D.E., Marktel, Sarah, Chiesa, Robert, Cowan, Morton J., Slatter, Mary A., Stricherz, Melisa K., Jennissen, Cathryn, Long-Boyle, Janel R., Boelens, Jaap Jan
Publicado no Lancet Haematol (2016)