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

BMN 250 is being developed as enzyme replacement therapy for Sanfilippo type B, a primarily neurological rare disease, in which patients have deficient lysosomal alpha-N-acetylglucosaminidase (NAGLU) enzyme activity. BMN 250 is taken up in target cells by the cation-independent mannose 6-phosphate r...

תיאור מלא

שמור ב:
מידע ביבליוגרפי
הוצא לאור ב:Drug Deliv Transl Res
Main Authors: Grover, 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, Anil
פורמט: Artigo
שפה:Inglês
יצא לאור: Springer US 2020
נושאים:
Original Article
גישה מקוונת:https://ncbi.nlm.nih.gov/pmc/articles/PMC7066106/
https://ncbi.nlm.nih.gov/pubmed/31942701
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s13346-019-00683-6
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