未登录 分享 收藏
分享 收藏
分享 收藏
分享 收藏
分享 收藏
分享 收藏
Systemic immune challenge exacerbates neurodegeneration in a model of neurological lysosomal disease Mandolfo, 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. 分享 收藏
Improving newborn screening test performance for metachromatic leukodystrophy: Recommendation from a pre-pilot study that identified a late-infantile case for treatment Wu, 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. 分享 收藏
Systemic inflammation leads to neuronal loss and exacerbates behavioural deficits in a mouse model of MPS IIIA Mandolfo, 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 分享 收藏
Clinical outcomes and sustained biochemical engraftment following ex-vivo autologous stem cell gene therapy for mucopolysaccharidosis type IIIA Jones, 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 分享 收藏
A UK-based pre-pilot newborn screening study for metachromatic leukodytrosphy identified a late infantile case Wu, Teresa Hoi-Yee; Brown, Heather; Church, Heather J.; Hutton, Rebekah; Ram, Dipak; Kershaw, Christopher; Chanson, Charlotte; Henderson, Michael J.; Bigger, Brian; Jones, Simon 分享 收藏
RETRACTION: An Innovative Tool for Evidence-Based, Personalized Treatment Trials in Mucopolysaccharidosis (Retraction of Vol 15, art no 1565, 2023) Wiesinger, Anna-Maria; Bigger, Brian; Giugliani, Roberto; Lampe, Christina; Scarpa, Maurizio; Moser, Tobias; Kampmann, Christoph; Zimmermann, Georg; Lagler, Florian B. 分享 收藏
分享 收藏
Intraparenchymal convection enhanced delivery of AAV in sheep to treat Mucopolysaccharidosis IIIC O'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. 分享 收藏
RETRACTED: An Innovative Tool for Evidence-Based, Personalized Treatment Trials in Mucopolysaccharidosis (Retracted Article) Wiesinger, Anna-Maria; Bigger, Brian; Giugliani, Roberto; Lampe, Christina; Scarpa, Maurizio; Moser, Tobias; Kampmann, Christoph; Zimmermann, Georg; Lagler, Florian B. 分享 收藏
分享 收藏
Sustained biochemical engraftment and early clinical outcomes following ex-vivo autologous stem cell gene therapy for mucopolysaccharidosis type IIIA Jones, 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 分享 收藏
Identification of novel peptide sequences to fuse to iduronate-2-sulfatase for the treatment of Hunter syndrome Wood, Shaun R.; Chaudrhy, Ahsan; Ellison, Stuart M.; Searle, Rachel; Forte, Gabriella; O'Leary, Claire; Gleitz, Helene; Liao, Aiyin; Holley, Rebecca; Bigger, Brian 分享 收藏
分享 收藏
分享 收藏