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Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatment of Sanfilippo type B
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Artigo
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Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatment of Sanfilippo type B

Grover, Anita ; Crippen-Harmon, Danielle ; Nave, Lacey ; Vincelette, Jon ; Wait, Jill C. M. ; Melton, Andrew C. ; Lawrence, Roger ; Brown, Jillian R. ; Webster, Katherine A. ; Yip, Bryan K. ; Baridon, Brian ; Vitelli, Catherine ; Rigney, Sara ; Christianson, Terri M. ; Tiger, Pascale M. N. ; Lo, Melanie J. ; Holtzinger, John ; Shaywitz, Adam J. ; Crawford, Brett E. ; Fitzpatrick, Paul A. ; LeBowitz, Jonathan H. ; Bullens, Sherry ; Aoyagi-Scharber, Mika ; Bunting, Stuart ; O’Neill, Charles A. ; Pinkstaff, Jason ; Bagri, Anil

Drug delivery and translational research, 2020-04, Vol.10 (2), p.425-439 [Periódico revisado por pares]

New York: Springer US

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2
Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB
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Artigo
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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 [Periódico revisado por pares]

United States: National Academy of Sciences

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3
BMN 250, a fusion of lysosomal alpha-N-acetylglucosaminidase with IGF2, exhibits different patterns of cellular uptake into critical cell types of Sanfilippo syndrome B disease pathogenesis
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BMN 250, a fusion of lysosomal alpha-N-acetylglucosaminidase with IGF2, exhibits different patterns of cellular uptake into critical cell types of Sanfilippo syndrome B disease pathogenesis

Yogalingam, Gouri ; Luu, Amanda R ; Prill, Heather ; Lo, Melanie J ; Yip, Bryan ; Holtzinger, John ; Christianson, Terri ; Aoyagi-Scharber, Mika ; Lawrence, Roger ; Crawford, Brett E ; LeBowitz, Jonathan H Dardis, Andrea

PloS one, 2019-01, Vol.14 (1), p.e0207836-e0207836 [Periódico revisado por pares]

United States: Public Library of Science

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