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1 |
Material Type: Artigo
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Engineering of a recombinant NAGLU fusion protein with insulin-like growth factor 2 leads to improved cellular uptake via a glycosylation-independent lysosomal targeting pathwayAoyagi-Scharber, Mika ; Christianson, Terri ; Wendt, Daniel J. ; Tiger, Pascale M.N. ; Yip, Bryan K. ; Holtzinger, John ; Chen, Zhi ; Woloszynek, Josh ; Cheung, Diana S. ; Lo, Melanie J. ; Dickson, Patricia ; Fitzpatrick, Paul A. ; LeBowitz, Jonathan H.Molecular genetics and metabolism, 2014-02, Vol.111 (2), p.S20-S20 [Periódico revisado por pares]Elsevier IncTexto completo disponível |
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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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Time- and dose-dependent normalization of pathological lysosomal storage and biochemistry in the mucopolysaccharidosis ΙΙΙΒ (MPS ΙΙΙΒ, Sanfilippo syndrome type Β) mouse model by intracerebroventricular enzyme replacement therapy with ΒΜΝ 250, a ΝAGLU-ΙGF2 fusion proAoyagi-Scharber, Mika ; Vincelette, Jon ; Lawrence, Roger ; Crippen-Harmon, Danielle ; Yip, Bryan K. ; Baridon, Brian ; Prill, Heather ; Minto, Wesley C. ; Van Vleet, Jeremy L. ; Vitelli, Catherine ; Adintori, Evan G. ; Christianson, Terri ; Tiger, Pascale M.N. ; Lo, Melanie J. ; Holtzinger, John ; Chen, Eric ; Webster, Katherine A. ; Brown, Jillian R. ; Fitzpatrick, Paul A. ; LeBowitz, Jonathan H. ; Bagri, Anil ; Bullens, Sherry ; Crawford, Brett E. ; Bunting, StuartMolecular genetics and metabolism, 2016-02, Vol.117 (2), p.S21-S21 [Periódico revisado por pares]Elsevier IncTexto completo disponível |
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Material Type: Artigo
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Histologic characterization of the progression of central nervous system pathology in the mucopolysaccharidosis IIIB (MPS IIIB, Sanfilippo syndrome type B) mouse model and bio-distribution and efficacy of the intracerebroventricular enzyme replacement therapy, BMN 250Bagri, Anil ; Harmon, Danielle ; Vincelette, Jon ; Vitelli, Catherine ; Minto, Wesley ; Yates, Bridget ; Rigney, Sara ; Santiago, Pam ; Baridon, Brian ; Xie, Lin ; Gorostiza, Olivia ; Christianson, Terri ; Tiger, Pascale M.N. ; Yip, Bryan K. ; Lo, Melanie J. ; Holtzinger, John ; Chen, Eric ; Aoyagi-Scharber, Mika ; Fitzpatrick, Paul ; LeBowitz, Jonathan H. ; Bullens, Sherry ; Bunting, StuartMolecular genetics and metabolism, 2016-02, Vol.117 (2), p.S23-S24 [Periódico revisado por pares]Elsevier IncTexto completo disponível |
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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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Material Type: Artigo
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The operation of a multiple stockroomHoltzinger, Albert HJournal of chemical education, 1953-10, Vol.30 (10), p.512 [Periódico revisado por pares]Division of Chemical EducationTexto completo disponível |
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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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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 |
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Material Type: Artigo
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WEB NECROSIS IN MUTE SWANS—A REPORT OF AN OUTBREAKYATES, VANCE J. ; MILLER, LOUISE T. ; JASTY, V. ; WILLEY, CHARLES H. ; HOLTZINGER, MARIANBulletin of the Wildlife Disease Association, 1969-01, Vol.5 (1), p.33-34Wildlife Disease AssociationTexto 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 |