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1 |
Material Type: Artigo
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Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B MiceAoyagi-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, StuartMolecular therapy. Methods & clinical development, 2017-09, Vol.6 (C), p.43-53 [Periódico revisado por pares]United States: Elsevier LimitedTexto completo disponível |
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Material Type: Artigo
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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 pathogenesisYogalingam, 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, AndreaPloS one, 2019-01, Vol.14 (1), p.e0207836-e0207836 [Periódico revisado por pares]United States: Public Library of ScienceTexto completo disponível |
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Material Type: Artigo
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Differential Uptake of NAGLU-IGF2 and Unmodified NAGLU in Cellular Models of Sanfilippo Syndrome Type BPrill, 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, RogerMolecular therapy. Methods & clinical development, 2019-09, Vol.14, p.56-63 [Periódico revisado por pares]United States: Elsevier IncTexto completo disponível |