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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: parker h. 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.
Innate Immunity in Mucopolysaccharide Diseases.
Mandolfo O, Parker H, Bigger B. Mandolfo O, et al. Among authors: parker h. Int J Mol Sci. 2022 Feb 11;23(4):1999. doi: 10.3390/ijms23041999. Int J Mol Sci. 2022. PMID: 35216110 Free PMC article. Review.
High dose genistein in Sanfilippo syndrome: A randomised controlled trial.
Ghosh A, Rust S, Langford-Smith K, Weisberg D, Canal M, Breen C, Hepburn M, Tylee K, Vaz FM, Vail A, Wijburg F, O'Leary C, Parker H, Wraith JE, Bigger BW, Jones SA. Ghosh A, et al. Among authors: parker h. J Inherit Metab Dis. 2021 Sep;44(5):1248-1262. doi: 10.1002/jimd.12407. Epub 2021 Jun 13. J Inherit Metab Dis. 2021. PMID: 34047372 Clinical Trial.
A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency.
Tordo J, O'Leary C, Antunes ASLM, Palomar N, Aldrin-Kirk P, Basche M, Bennett A, D'Souza Z, Gleitz H, Godwin A, Holley RJ, Parker H, Liao AY, Rouse P, Youshani AS, Dridi L, Martins C, Levade T, Stacey KB, Davis DM, Dyer A, Clément N, Björklund T, Ali RR, Agbandje-McKenna M, Rahim AA, Pshezhetsky A, Waddington SN, Linden RM, Bigger BW, Henckaerts E. Tordo J, et al. Among authors: parker h. Brain. 2018 Jul 1;141(7):2014-2031. doi: 10.1093/brain/awy126. Brain. 2018. PMID: 29788236 Free PMC article.
814 results