Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB

Kan, Shih-hsin ; Aoyagi-Scharber, Mika ; Le, Steven Q. ; Vincelette, Jon ; Ohmi, Kazuhiro ; Bullens, Sherry ; Wendt, Daniel J. ; Christiansen, Terri M. ; Tiger, Pascale M. N. ; Brown, Julian R. ; Lawrence, Roger ; Yip, Bryan K. ; Holtzinger, John ; Bagri, Anil ; Crippen-Harmon, Danielle ; Vondrak, Kristen N. ; Chen, Zhi ; Hague, Chuck M. ; Woloszynek, Josh C. ; Cheung, Diana S. ; Webster, Katherine A. ; Adintori, Evan G. ; Lo, Melanie J. ; Wong, Wesley ; Fitzpatrick, Paul A. ; LeBowitz, Jonathan H. ; Crawford, Brett E. ; Bunting, Stuart ; Dickson, Patricia I. ; Neufeld, Elizabeth F.

Proceedings of the National Academy of Sciences - PNAS, 2014-10, Vol.111 (41), p.14870-14875 [Peer Reviewed Journal]

United States: National Academy of Sciences

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