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Design and validation of a GMP stem cell manufacturing protocol for MPSII hematopoietic stem cell gene therapy.
Ellison S, Buckland K, Learmonth Y, Day V, Kalra S, Howe L, Roman-Rodriguez FJ, Bonafont J, Booth L, Holley R, Smythe J, Jones S, Thrasher A, Booth C, Bigger BW. Ellison S, et al. Mol Ther Methods Clin Dev. 2024 May 21;32(2):101271. doi: 10.1016/j.omtm.2024.101271. eCollection 2024 Jun 13. Mol Ther Methods Clin Dev. 2024. PMID: 38946936 Free PMC article.
Recommendations for the management of MPS VI: systematic evidence- and consensus-based guidance.
Akyol MU, Alden TD, Amartino H, Ashworth J, Belani K, Berger KI, Borgo A, Braunlin E, Eto Y, Gold JI, Jester A, Jones SA, Karsli C, Mackenzie W, Marinho DR, McFadyen A, McGill J, Mitchell JJ, Muenzer J, Okuyama T, Orchard PJ, Stevens B, Thomas S, Walker R, Wynn R, Giugliani R, Harmatz P, Hendriksz C, Scarpa M; MPS Consensus Programme Steering Committee; MPS Consensus Programme Co-Chairs. Akyol MU, et al. Among authors: jones sa. Orphanet J Rare Dis. 2019 May 29;14(1):118. doi: 10.1186/s13023-019-1080-y. Orphanet J Rare Dis. 2019. PMID: 31142378 Free PMC article.
Acid sphingomyelinase deficiency and Gaucher disease: Underdiagnosed and often treatable causes of hepatomegaly, splenomegaly, and low HDL cholesterol in lean individuals.
Mistry PK, Cassiman D, Jones SA, Lachmann R, Lukina E, Prada CE, Wasserstein MP, Thurberg BL, Foster MC, Patel RM, Underhill LH, Peterschmitt MJ. Mistry PK, et al. Among authors: jones sa. Hepatol Commun. 2025 Jan 7;9(1):e0621. doi: 10.1097/HC9.0000000000000621. eCollection 2025 Jan 1. Hepatol Commun. 2025. PMID: 39774103 Free PMC article.
939 results