Gene-modified neural progenitor cells for the treatment of neuropathic lysosomal storage diseases by Oriana Mandolfo, Brian W Bigger

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Delivering gene therapy for mucopolysaccharide diseases by Shaun R. Wood, Brian W. Bigger

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Current and Future Treatment of Mucopolysaccharidosis (MPS) Type II: Is Brain-Targeted Stem Cell Gene Therapy the Solution for This Devastating Disorder? by Claire Horgan, Simon A. Jones, Brian W. Bigger, Robert Wynn

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Identification of age-dependent motor and neuropsychological behavioural abnormalities in a mouse model of Mucopolysaccharidosis Type II. by Hélène F E Gleitz, Claire O'Leary, Rebecca J Holley, Brian W Bigger

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Substrate accumulation and extracellular matrix remodelling promote persistent upper airway disease in mucopolysaccharidosis patients on enzyme replacement therapy. by Abhijit Ricky Pal, Jean Mercer, Simon A Jones, Iain A Bruce, Brian W Bigger

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Strategies for the Induction of Immune Tolerance to Enzyme Replacement Therapy in Mucopolysaccharidosis Type I by Arunabha Ghosh, Aiyin Liao, Claire O’Leary, Jean Mercer, Karen Tylee, Anu Goenka, Rebecca Holley, Simon A. Jones, Brian W. Bigger

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

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Sustained long-term disease correction in a murine model of MPSII following stem cell gene therapy by Stuart Ellison, Aiyin Liao, Hélène F.E. Gleitz, Helen Parker, Laura Booth, John Robinson, Shaun Wood, Jessica Taylor, Rebecca Holley, Brian W. Bigger

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

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Non-myeloablative busulfan chimeric mouse models are less pro-inflammatory than head-shielded irradiation for studying immune cell interactions in brain tumours by A. Saam Youshani, Samuel Rowlston, Claire O’Leary, Gabriella Forte, Helen Parker, Aiyin Liao, Brian Telfer, Kaye Williams, Ian D. Kamaly-Asl, Brian W. Bigger

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Brain‐targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanisms by Hélène FE Gleitz, Ai Yin Liao, James R Cook, Samuel F Rowlston, Gabriella MA Forte, Zelpha D'Souza, Claire O'Leary, Rebecca J Holley, Brian W Bigger

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Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III by Arunabha Ghosh, Elsa Shapiro, Stewart Rust, Kathleen Delaney, Samantha Parker, Adam J Shaywitz, Adelaida Morte, Gillian Bubb, Maureen Cleary, Tien Bo, Christine Lavery, Brian W Bigger, Simon A Jones

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Haematopoietic stem cell gene therapy with IL‐1Ra rescues cognitive loss in mucopolysaccharidosis IIIA by Helen Parker, Stuart M Ellison, Rebecca J Holley, Claire O'Leary, Aiyin Liao, Jalal Asadi, Emily Glover, Arunabha Ghosh, Simon Jones, Fiona L Wilkinson, David Brough, Emmanuel Pinteaux, Hervé Boutin, Brian W Bigger

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Genistein improves neuropathology and corrects behaviour in a mouse model of neurodegenerative metabolic disease. by Marcelina Malinowska, Fiona L Wilkinson, Kia J Langford-Smith, Alex Langford-Smith, Jillian R Brown, Brett E Crawford, Marie T Vanier, Grzegorz Grynkiewicz, Rob F Wynn, J Ed Wraith, Grzegorz Wegrzyn, Brian W Bigger

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Obstructive Sleep Apnea in MPS by Abhijit Ricky Pal MBBChir, MA, MD, FRCS(ORL-HNS), Nailah Brown BSc, MPhil, Simon A. Jones MB ChB, MRCPCH, Brian W. Bigger PhD, Iain A. Bruce MB ChB, MD, FRCS(ORL-HNS)

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Neuropathology in mouse models of mucopolysaccharidosis type I, IIIA and IIIB. by Fiona L Wilkinson, Rebecca J Holley, Kia J Langford-Smith, Soumya Badrinath, Aiyin Liao, Alex Langford-Smith, Jonathan D Cooper, Simon A Jones, J Ed Wraith, Rob F Wynn, Catherine L R Merry, Brian W Bigger

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Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Ex Vivo Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA by Stuart M. Ellison, Aiyin Liao, Shaun Wood, Jessica Taylor, Amir Saam Youshani, Sam Rowlston, Helen Parker, Myriam Armant, Alessandra Biffi, Lucas Chan, Farzin Farzaneh, Rob Wynn, Simon A. Jones, Paul Heal, H. Bobby Gaspar, Brian W. Bigger

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High content screening of patient-derived cell lines highlights the potential of non-standard chemotherapeutic agents for the treatment of glioblastoma. by Kenny Kwok-Hei Yu, Jessica T Taylor, Omar N Pathmanaban, Amir Saam Youshani, Deniz Beyit, Joanna Dutko-Gwozdz, Roderick Benson, Gareth Griffiths, Ian Peers, Peter Cueppens, Brian A Telfer, Kaye J Williams, Catherine McBain, Ian D Kamaly-Asl, Brian W Bigger

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Intraparenchymal convection enhanced delivery of AAV in sheep to treat Mucopolysaccharidosis IIIC by Claire O’Leary, Gabriella Forte, Nadia L. Mitchell, Amir Saam Youshani, Adam Dyer, Martin P. Wellby, Katharina N. Russell, Samantha J. Murray, Nelly Jolinon, Simon A Jones, Kevin Stacey, Daniel M. Davis, Els Henckaerts, David N. Palmer, Ian Kamaly-Asl, Brian W. Bigger

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

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