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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: bigger bw. 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. Among authors: bigger bw. 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.
Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler's syndrome after pharmacological enzyme replacement therapy.
Saif MA, Bigger BW, Brookes KE, Mercer J, Tylee KL, Church HJ, Bonney DK, Jones S, Wraith JE, Wynn RF. Saif MA, et al. Among authors: bigger bw. Haematologica. 2012 Sep;97(9):1320-8. doi: 10.3324/haematol.2011.058644. Epub 2012 Feb 27. Haematologica. 2012. PMID: 22371174 Free PMC article.
106 results