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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.
Grover A, Crippen-Harmon D, Nave L, Vincelette J, Wait JCM, Melton AC, Lawrence R, Brown JR, Webster KA, Yip BK, Baridon B, Vitelli C, Rigney S, Christianson TM, Tiger PMN, Lo MJ, Holtzinger J, Shaywitz AJ, Crawford BE, Fitzpatrick PA, LeBowitz JH, Bullens S, Aoyagi-Scharber M, Bunting S, O'Neill CA, Pinkstaff J, Bagri A. Grover A, et al. Among authors: holtzinger j. Drug Deliv Transl Res. 2020 Apr;10(2):425-439. doi: 10.1007/s13346-019-00683-6. Drug Deliv Transl Res. 2020. PMID: 31942701 Free PMC article.
Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB.
Kan SH, Aoyagi-Scharber M, Le SQ, Vincelette J, Ohmi K, Bullens S, Wendt DJ, Christianson TM, Tiger PM, Brown JR, Lawrence R, Yip BK, Holtzinger J, Bagri A, Crippen-Harmon D, Vondrak KN, Chen Z, Hague CM, Woloszynek JC, Cheung DS, Webster KA, Adintori EG, Lo MJ, Wong W, Fitzpatrick PA, LeBowitz JH, Crawford BE, Bunting S, Dickson PI, Neufeld EF. Kan SH, et al. Among authors: holtzinger j. Proc Natl Acad Sci U S A. 2014 Oct 14;111(41):14870-5. doi: 10.1073/pnas.1416660111. Epub 2014 Sep 29. Proc Natl Acad Sci U S A. 2014. PMID: 25267636 Free PMC article.
Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice.
Aoyagi-Scharber M, Crippen-Harmon D, Lawrence R, Vincelette J, Yogalingam G, Prill H, Yip BK, Baridon B, Vitelli C, Lee A, Gorostiza O, Adintori EG, Minto WC, Van Vleet JL, Yates B, Rigney S, Christianson TM, Tiger PMN, Lo MJ, Holtzinger J, Fitzpatrick PA, LeBowitz JH, Bullens S, Crawford BE, Bunting S. Aoyagi-Scharber M, et al. Among authors: holtzinger j. Mol Ther Methods Clin Dev. 2017 Jun 6;6:43-53. doi: 10.1016/j.omtm.2017.05.009. eCollection 2017 Sep 15. Mol Ther Methods Clin Dev. 2017. PMID: 28664165 Free PMC article.
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 pathogenesis.
Yogalingam G, Luu AR, Prill H, Lo MJ, Yip B, Holtzinger J, Christianson T, Aoyagi-Scharber M, Lawrence R, Crawford BE, LeBowitz JH. Yogalingam G, et al. Among authors: holtzinger j. PLoS One. 2019 Jan 18;14(1):e0207836. doi: 10.1371/journal.pone.0207836. eCollection 2019. PLoS One. 2019. PMID: 30657762 Free PMC article.