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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. J Clin Invest. 2022 Oct 17;132(20):e163107. doi: 10.1172/JCI163107. J Clin Invest. 2022. PMID: 36040802 Free PMC article.