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Cross-species efficacy of enzyme replacement therapy for CLN1 disease in mice and sheep.
Nelvagal HR, Eaton SL, Wang SH, Eultgen EM, Takahashi K, Le SQ, Nesbitt R, Dearborn JT, Siano N, Puhl AC, Dickson PI, Thompson G, Murdoch F, Brennan PM, Gray M, Greenhalgh SN, Tennant P, Gregson R, Clutton E, Nixon J, Proudfoot C, Guido S, Lillico SG, Whitelaw CBA, Lu JY, Hofmann SL, Ekins S, Sands MS, Wishart TM, Cooper JD. Nelvagal HR, et al. Among authors: le sq. J Clin Invest. 2022 Oct 17;132(20):e163107. doi: 10.1172/JCI163107. J Clin Invest. 2022. PMID: 36040802 Free PMC article.
Early versus late treatment of spinal cord compression with long-term intrathecal enzyme replacement therapy in canine mucopolysaccharidosis type I.
Dickson PI, Hanson S, McEntee MF, Vite CH, Vogler CA, Mlikotic A, Chen AH, Ponder KP, Haskins ME, Tippin BL, Le SQ, Passage MB, Guerra C, Dierenfeld A, Jens J, Snella E, Kan SH, Ellinwood NM. Dickson PI, et al. Among authors: le sq. Mol Genet Metab. 2010 Oct-Nov;101(2-3):115-22. doi: 10.1016/j.ymgme.2010.06.020. Epub 2010 Jul 23. Mol Genet Metab. 2010. PMID: 20655780 Free PMC article.
60 results