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Galactosialidosis: preclinical enzyme replacement therapy in a mouse model of the disease, a proof of concept.
Cadaoas J, Hu H, Boyle G, Gomero E, Mosca R, Jayashankar K, Machado M, Cullen S, Guzman B, van de Vlekkert D, Annunziata I, Vellard M, Kakkis E, Koppaka V, d'Azzo A. Cadaoas J, et al. Among authors: vellard m. Mol Ther Methods Clin Dev. 2020 Nov 20;20:191-203. doi: 10.1016/j.omtm.2020.11.012. eCollection 2021 Mar 12. Mol Ther Methods Clin Dev. 2020. PMID: 33426146 Free PMC article.
Lipoprotein receptor binding, cellular uptake, and lysosomal delivery of fusions between the receptor-associated protein (RAP) and alpha-L-iduronidase or acid alpha-glucosidase.
Prince WS, McCormick LM, Wendt DJ, Fitzpatrick PA, Schwartz KL, Aguilera AI, Koppaka V, Christianson TM, Vellard MC, Pavloff N, Lemontt JF, Qin M, Starr CM, Bu G, Zankel TC. Prince WS, et al. Among authors: vellard mc. J Biol Chem. 2004 Aug 13;279(33):35037-46. doi: 10.1074/jbc.M402630200. Epub 2004 May 31. J Biol Chem. 2004. PMID: 15170390 Free article.
19 results