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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: hofmann sl. J Clin Invest. 2022 Oct 17;132(20):e163107. doi: 10.1172/JCI163107. J Clin Invest. 2022. PMID: 36040802 Free PMC article.
Intravenous high-dose enzyme replacement therapy with recombinant palmitoyl-protein thioesterase reduces visceral lysosomal storage and modestly prolongs survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis.
Hu J, Lu JY, Wong AM, Hynan LS, Birnbaum SG, Yilmaz DS, Streit BM, Lenartowicz EM, Thompson TC, Cooper JD, Hofmann SL. Hu J, et al. Among authors: hofmann sl. Mol Genet Metab. 2012 Sep;107(1-2):213-21. doi: 10.1016/j.ymgme.2012.05.009. Epub 2012 May 22. Mol Genet Metab. 2012. PMID: 22704978 Free PMC article.
Histochemical localization of palmitoyl protein thioesterase-1 activity.
Dearborn JT, Ramachandran S, Shyng C, Lu JY, Thornton J, Hofmann SL, Sands MS. Dearborn JT, et al. Among authors: hofmann sl. Mol Genet Metab. 2016 Feb;117(2):210-6. doi: 10.1016/j.ymgme.2015.11.004. Epub 2015 Nov 11. Mol Genet Metab. 2016. PMID: 26597320 Free PMC article.
79 results