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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.
Ellison SM, Liao A, Wood S, Taylor J, Youshani AS, Rowlston S, Parker H, Armant M, Biffi A, Chan L, Farzaneh F, Wynn R, Jones SA, Heal P, Gaspar HB, Bigger BW. Ellison SM, et al. Among authors: jones sa. Mol Ther Methods Clin Dev. 2019 Apr 6;13:399-413. doi: 10.1016/j.omtm.2019.04.001. eCollection 2019 Jun 14. Mol Ther Methods Clin Dev. 2019. PMID: 31044143 Free PMC article.
Heparin cofactor II-thrombin complex and dermatan sulphate:chondroitin sulphate ratio are biomarkers of short- and long-term treatment effects in mucopolysaccharide diseases.
Langford-Smith KJ, Mercer J, Petty J, Tylee K, Church H, Roberts J, Moss G, Jones S, Wynn R, Wraith JE, Bigger BW. Langford-Smith KJ, et al. J Inherit Metab Dis. 2011 Apr;34(2):499-508. doi: 10.1007/s10545-010-9254-8. Epub 2010 Dec 18. J Inherit Metab Dis. 2011. PMID: 21170681 Free PMC article.
Neuropathology in mouse models of mucopolysaccharidosis type I, IIIA and IIIB.
Wilkinson FL, Holley RJ, Langford-Smith KJ, Badrinath S, Liao A, Langford-Smith A, Cooper JD, Jones SA, Wraith JE, Wynn RF, Merry CL, Bigger BW. Wilkinson FL, et al. Among authors: jones sa. PLoS One. 2012;7(4):e35787. doi: 10.1371/journal.pone.0035787. Epub 2012 Apr 27. PLoS One. 2012. PMID: 22558223 Free PMC article.
Assessment of sleep in children with mucopolysaccharidosis type III.
Mahon LV, Lomax M, Grant S, Cross E, Hare DJ, Wraith JE, Jones S, Bigger B, Langford-Smith K, Canal M. Mahon LV, et al. PLoS One. 2014 Feb 4;9(2):e84128. doi: 10.1371/journal.pone.0084128. eCollection 2014. PLoS One. 2014. PMID: 24504123 Free PMC article.
938 results