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1 |
Material Type: Artigo
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Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIBKan, 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 SciencesTexto completo disponível |
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2 |
Material Type: 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 BGrover, 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, AnilDrug delivery and translational research, 2020-04, Vol.10 (2), p.425-439 [Periódico revisado por pares]New York: Springer USTexto completo disponível |
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3 |
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 |