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Brian Bigger

University of Manchester

41H指数
207论文数
5.2K被引数
收录论文 102
发表时间
Clinical and biochemical correction of a patient with neurodegenerative Mucopolysaccharidosis IIIA using hematopoietic stem cell gene therapy使用造血干细胞基因疗法对神经退行性黏多糖贮积症IIIA患者进行临床和生化纠正
err2026-09-25
err0
PREAI
errJane L. Kinsella; Simon A. Jones; Karen F. Buckland; Rebecca J. Holley; Helena Lee; Wendy Ogden; Jane Potter; Heather J. Church; Kathryn L. Brammeier; Karen L. Tylee; Stuart Ellison; Rachel Searle; Claire Booth; Farzin Farzeneh; Louise Sherwin; Adrian J. Thrasher; Stewart Rust; Brian W. Bigger; Robert F. Wynn
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Human iPSC-derived neural progenitor gene therapy improves outcomes in a neuropathic lysosomal disease人源iPSC衍生的神经祖细胞基因疗法改善神经病理溶酶体疾病的结局
err2026-08-10
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errOAAI
errOriana Mandolfo; Yuko Ishikawa Learmonth; Asma’u Usman; Maryann Lorino; Tereza Andreou; Rebecca Holley; Bei Qiu; Tristan Mckay; Brian Bigger
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An evidence-based individual treatment trial with adalimumab in a patient with Morquio syndrome
err2025-02-01
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PREAI
errWiesinger, Anna-Maria; Bigger, Brian; Giugliani, Roberto; Lampe, Christina; Scarpa, Maurizio; Moser, Tobias; Zimmermann, Georg; Lagler, Florian
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Generation of a novel immunodeficient mouse model of Mucopolysaccharidosis type IIIA to test human stem cell-based therapies
err2024-09-01
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PREAI
errMandolfo, Oriana; Parker, Helen; Usman, Asma'u; Learmonth, Yuko Ishikawa; Holley, Rebecca J.; Macdonald, Andrew; Mckay, Tristan; Bigger, Brian
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Systemic immune challenge exacerbates neurodegeneration in a model of neurological lysosomal disease
err2024-06-18
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errOAAI
errMandolfo, Oriana; Parker, Helen; Aguado, Elia; Learmonth, Yuko Ishikawa; Liao, Ai Yin; O'Leary, Claire; Ellison, Stuart; Forte, Gabriella; Taylor, Jessica; Wood, Shaun; Searle, Rachel; Holley, Rebecca J.; Boutin, Herve; Bigger, Brian W.
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Improving newborn screening test performance for metachromatic leukodystrophy: Recommendation from a pre-pilot study that identified a late-infantile case for treatment
err2024-05-01
err4
errOAAI
errWu, Teresa H. Y.; Brown, Heather A.; Church, Heather J.; Kershaw, Christopher J.; Hutton, Rebekah; Egerton, Christine; Cooper, James; Tylee, Karen; Cohen, Rebecca N.; Gokhale, David; Ram, Dipak; Morton, Georgina; Henderson, Michael; Bigger, Brian W.; Jones, Simon A.
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Systemic inflammation leads to neuronal loss and exacerbates behavioural deficits in a mouse model of MPS IIIA
err2024-02-01
err0
PREAI
errMandolfo, Oriana; Parker, Helen; Aguado, Elia; Learmonth, Yuko Ishikawa; Liao, Aiyin; O'Leary, Claire; Ellison, Stuart M.; Taylor, Jessica; Wood, Shaun R.; Holley, Rebecca; Boutin, Herve; Bigger, Brian
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Clinical outcomes and sustained biochemical engraftment following ex-vivo autologous stem cell gene therapy for mucopolysaccharidosis type IIIA
err2024-02-01
err3
PREAI
errJones, Simon; Kinsella, Jane; Holley, Rebecca J.; Potter, Jane; Booth, Claire; Buckland, Karen; Rust, Stewart; Bromley, Rebecca; Church, Heather J.; Lee, Helena; Ford, Laura; Learmonth, Yuko Ishikawa; Searle, Rachel; Ellison, Stuart M.; Wynn, Robert; Bigger, Brian
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A UK-based pre-pilot newborn screening study for metachromatic leukodytrosphy identified a late infantile case
err2024-02-01
err0
PREAI
errWu, Teresa Hoi-Yee; Brown, Heather; Church, Heather J.; Hutton, Rebekah; Ram, Dipak; Kershaw, Christopher; Chanson, Charlotte; Henderson, Michael J.; Bigger, Brian; Jones, Simon
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RETRACTION: An Innovative Tool for Evidence-Based, Personalized Treatment Trials in Mucopolysaccharidosis (Retraction of Vol 15, art no 1565, 2023)
err2023-11-07
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errOAAI
errWiesinger, Anna-Maria; Bigger, Brian; Giugliani, Roberto; Lampe, Christina; Scarpa, Maurizio; Moser, Tobias; Kampmann, Christoph; Zimmermann, Georg; Lagler, Florian B.
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Intraparenchymal convection enhanced delivery of AAV in sheep to treat Mucopolysaccharidosis IIIC
err2023-07-05
err2
errOAAI
errO'Leary, Claire; Forte, Gabriella; Mitchell, Nadia L.; Youshani, Amir Saam; Dyer, Adam; Wellby, Martin P.; Russell, Katharina N.; Murray, Samantha J.; Jolinon, Nelly; Jones, Simon A.; Stacey, Kevin; Davis, Daniel M.; Henckaerts, Els; Palmer, David N.; Kamaly-Asl, Ian; Bigger, Brian W.
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RETRACTED: An Innovative Tool for Evidence-Based, Personalized Treatment Trials in Mucopolysaccharidosis (Retracted Article)
err2023-05-22
err3
errOAAI
errWiesinger, Anna-Maria; Bigger, Brian; Giugliani, Roberto; Lampe, Christina; Scarpa, Maurizio; Moser, Tobias; Kampmann, Christoph; Zimmermann, Georg; Lagler, Florian B.
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Developing an iPSC-based neural gene therapy approach for MPS IIIA
err2023-02-01
err0
PREAI
errMandolfo, Oriana; Learmonth, Yuko Ishikawa; Usman, Asma'u; Andreou, Tereza; Taylor, Jessica; Parker, Helen; McKay, Tristan; Bigger, Brian
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Sustained biochemical engraftment and early clinical outcomes following ex-vivo autologous stem cell gene therapy for mucopolysaccharidosis type IIIA
err2023-02-01
err2
PREAI
errJones, Simon; Kinsella, Jane; Holley, Rebecca; Potter, Jane; Booth, Claire; Buckland, Karen; Rust, Stewart; Church, Heather J.; Brammeier, Kathryn L.; Lee, Helena; Ford, Laura; Ellison, Stuart M.; Bigger, Brian; Wynn, Robert
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Identification of novel peptide sequences to fuse to iduronate-2-sulfatase for the treatment of Hunter syndrome
err2023-02-01
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PREAI
errWood, Shaun R.; Chaudrhy, Ahsan; Ellison, Stuart M.; Searle, Rachel; Forte, Gabriella; O'Leary, Claire; Gleitz, Helene; Liao, Aiyin; Holley, Rebecca; Bigger, Brian
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A precision medicine tool for high utilization and quality of individual treatment trials with immunomodulatory drugs in mucopolysaccharidosis
err2023-02-01
err0
PREAI
errWiesinger, Anna-Maria; Bigger, Brian; Lampe, Christina; Giugliani, Roberto; Kampmann, Christoph; Moser, Tobias; Scarpa, Maurizio; Lagler, Florian B.
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Validation of a GMP stem cell gene therapy manufacturing process for mucopolysaccharidosis type II (MPS II) in preparation for an approved phase I/II clinical trial
err2023-02-01
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PREAI
errEllison, Stuart M.; Buckland, Karen; Ishikawalearmonth, Yuko; Booth, Claire; Wynn, Robert; Jones, Simon; Bigger, Brian
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