Art

J-GLOBAL ID：202002269317577315   Reference number：20A0716549

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

Sanfilippo型Bの治療のための酵素補充療法,BMN250に対するCNS細胞体内分布に対する静脈内および脳室内投与経路の翻訳研究【JST・京大機械翻訳】

Author (27)： Grover Anita (BioMarin Pharmaceutical Inc., Novato, CA, USA) ,  Crippen-Harmon Danielle (BioMarin Pharmaceutical Inc., Novato, CA, USA) ,  Nave Lacey (BioMarin Pharmaceutical Inc., Novato, CA, USA) ,  Vincelette Jon (BioMarin Pharmaceutical Inc., Novato, CA, USA) ,  Wait Jill C. M. (BioMarin Pharmaceutical Inc., Novato, CA, USA) ,  Melton Andrew C. (BioMarin Pharmaceutical Inc., Novato, CA, USA) ,  Lawrence Roger (BioMarin Pharmaceutical Inc., Novato, CA, USA) ,  Brown Jillian R. (BioMarin Pharmaceutical Inc., Novato, CA, USA) ,  Webster Katherine A. (BioMarin Pharmaceutical Inc., Novato, CA, USA) ,  Yip Bryan K. (BioMarin Pharmaceutical Inc., Novato, CA, USA) ,  Baridon Brian (BioMarin Pharmaceutical Inc., Novato, CA, USA) ,  Vitelli Catherine (BioMarin Pharmaceutical Inc., Novato, CA, USA) ,  Rigney Sara (BioMarin Pharmaceutical Inc., Novato, CA, USA) ,  Christianson Terri M. (BioMarin Pharmaceutical Inc., Novato, CA, USA) ,  Tiger Pascale M. N. (BioMarin Pharmaceutical Inc., Novato, CA, USA) ,  Lo Melanie J. (BioMarin Pharmaceutical Inc., Novato, CA, USA) ,  Holtzinger John (BioMarin Pharmaceutical Inc., Novato, CA, USA) ,  Shaywitz Adam J. (BioMarin Pharmaceutical Inc., Novato, CA, USA) ,  Crawford Brett E. (BioMarin Pharmaceutical Inc., Novato, CA, USA) ,  Fitzpatrick Paul A. (BioMarin Pharmaceutical Inc., Novato, CA, USA) ,  LeBowitz Jonathan H. (BioMarin Pharmaceutical Inc., Novato, CA, USA) ,  Bullens Sherry (BioMarin Pharmaceutical Inc., Novato, CA, USA) ,  Aoyagi-Scharber Mika (BioMarin Pharmaceutical Inc., Novato, CA, USA) ,  Bunting Stuart (BioMarin Pharmaceutical Inc., Novato, CA, USA) ,  O'Neill Charles A. (BioMarin Pharmaceutical Inc., Novato, CA, USA) ,  Pinkstaff Jason (BioMarin Pharmaceutical Inc., Novato, CA, USA) ,  Bagri Anil (BioMarin Pharmaceutical Inc., Novato, CA, USA)
Material： Drug Delivery and Translational Research
Volume： 10  Issue： 2  Page： 425-439  Publication year： 2020 
JST Material Number： W4295A  ISSN： 2190-393X  Document type： Article
Article type： 原著論文  Country of issue： Germany, Federal Republic of (DEU)  Language： ENGLISH (EN)

Abstract/Point： Abstract: BMN 250 is being dev...

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Thesaurus term： puberty(human) ,  cerebral vessel ,  clearance ,  receptor ,  human(primates) ,  Primates ,  neuron ,  pharmacology ,  blood-brain barrier ,  *cerebral ventricles ,  *biodistribution ,  *vein ,  lysosome ,  *enzyme ,  endothelial cell

Author keywords (6)： Lysosomal storage diseases ,  Enzyme replacement therapy ,  Sanfilippo type B ,  Alpha-N-acetylglucosaminidase ,  Mucopolysaccharidosis type III ,  Intracerebroventricular delivery

JST classification (2)： Enzyme in general  (EB09010D) ,  Metabolic diseases,nutritional diseases in general.  (GD05010J)

Terms in the title (9)： 治療 ,  酵素補充療法 ,  細胞 ,  体内分布 ,  静脈 ,  脳室内投与 ,  経路 ,  翻訳 ,  研究