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-论文-万方医学网

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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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第一作者： Grover, Anita

作者： Grover, Anita ^[1] ; 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

作者单位： BioMarin Pharmaceut Inc,BioMarin Pharmaceut Inc, 105 Digital Dr, Novato, CA 94949 USA ^[1]

关键词 Lysosomal storage diseases Enzyme replacement therapy Sanfilippo type B Alpha-N-acetylglucosaminidase Mucopolysaccharidosis type III Intracerebroventricular delivery BLOOD-BRAIN-BARRIER GROWTH-FACTOR-II MANNOSE 6-PHOSPHATE RECEPTOR DEVELOPMENTAL EXPRESSION MEDIATED TRANSPORT HEPARAN-SULFATE MOUSE MODEL GLIAL SCAR RAT SULFAMIDASE

发布时间 2022-06-23

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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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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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