Cross-species efficacy of enzyme replacement therapy for CLN1 disease in mice and sheep

Hemanth R. Nelvagal, Sam Eaton, Sophie H. Wang, Elizabeth M. Eultgen, Keigo Takahashi, Steven Q. Le, Rachel Nesbitt, Joshua T. Dearborn, Nicholas Siano, Ana C. Puhl, Patricia I. Dickson, Gerard Thompson, Fraser Murdoch, Paul Brennan, Mark Gray, Stephen Greenhalgh, Peter Tennant, Rachael Gregson, Eddie Clutton, James NixonChris Proudfoot, Stefano Guido, Simon Lillico, Bruce Whitelaw, Jui-Yun Lu, Sandra L Hofmann, Sean Ekins, Mark S. Sands , Thomas Wishart, Jonathan D Cooper

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Medicine & Life Sciences