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Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice
Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice
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Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice

Aoyagi-Scharber, Mika ; Crippen-Harmon, Danielle ; Lawrence, Roger ; Vincelette, Jon ; Yogalingam, Gouri ; Prill, Heather ; Yip, Bryan K ; Baridon, Brian ; Vitelli, Catherine ; Lee, Amanda ; Gorostiza, Olivia ; Adintori, Evan G ; Minto, Wesley C ; Van Vleet, Jeremy L ; Yates, Bridget ; Rigney, Sara ; Christianson, Terri M ; Tiger, Pascale M N ; Lo, Melanie J ; Holtzinger, John ; Fitzpatrick, Paul A ; LeBowitz, Jonathan H ; Bullens, Sherry ; Crawford, Brett E ; Bunting, Stuart

Molecular therapy. Methods & clinical development, 2017-09, Vol.6 (C), p.43-53 [Revista revisada por pares]

United States: Elsevier Limited

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

United States: National Academy of Sciences

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3
Differential Uptake of NAGLU-IGF2 and Unmodified NAGLU in Cellular Models of Sanfilippo Syndrome Type B
Differential Uptake of NAGLU-IGF2 and Unmodified NAGLU in Cellular Models of Sanfilippo Syndrome Type B
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Differential Uptake of NAGLU-IGF2 and Unmodified NAGLU in Cellular Models of Sanfilippo Syndrome Type B

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

Molecular therapy. Methods & clinical development, 2019-09, Vol.14, p.56-63 [Revista revisada por pares]

United States: Elsevier Inc

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4
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
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 [Revista revisada por pares]

United States: Public Library of Science

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