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Page 1
Intracerebroventricular administration of a modified hexosaminidase ameliorates late-stage neurodegeneration in a GM2 mouse model.
Lopez ME, Wendt D, Lawrence R, Gong K, Ong H, Yip B, Chen J, Mangini L, Handyside B, Giaramita A, Lamichhane A, Lo M, Agrawal V, Van Vleet J, Abolhesn A, Felix JB, Villalpando I, Bhat V, De Angelis R, Ru Y, Khan A, Fong S, Christianson T, Bullens S, Crawford BE, Bunting S, Aoyagi-Scharber M. Lopez ME, et al. Among authors: aoyagi scharber m. PLoS One. 2025 Jan 3;20(1):e0315005. doi: 10.1371/journal.pone.0315005. eCollection 2025. PLoS One. 2025. PMID: 39752451 Free PMC article.
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: aoyagi scharber m. 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.
Differential Uptake of NAGLU-IGF2 and Unmodified NAGLU in Cellular Models of Sanfilippo Syndrome Type B.
Prill H, Luu A, Yip B, Holtzinger J, Lo MJ, Christianson TM, Yogalingam G, Aoyagi-Scharber M, LeBowitz JH, Crawford BE, Lawrence R. Prill H, et al. Among authors: aoyagi scharber m. Mol Ther Methods Clin Dev. 2019 May 24;14:56-63. doi: 10.1016/j.omtm.2019.05.008. eCollection 2019 Sep 13. Mol Ther Methods Clin Dev. 2019. PMID: 31309128 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: aoyagi scharber m. PLoS One. 2019 Jan 18;14(1):e0207836. doi: 10.1371/journal.pone.0207836. eCollection 2019. PLoS One. 2019. PMID: 30657762 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. 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.
Discovery and Characterization of (8S,9R)-5-Fluoro-8-(4-fluorophenyl)-9-(1-methyl-1H-1,2,4-triazol-5-yl)-2,7,8,9-tetrahydro-3H-pyrido[4,3,2-de]phthalazin-3-one (BMN 673, Talazoparib), a Novel, Highly Potent, and Orally Efficacious Poly(ADP-ribose) Polymerase-1/2 Inhibitor, as an Anticancer Agent.
Wang B, Chu D, Feng Y, Shen Y, Aoyagi-Scharber M, Post LE. Wang B, et al. Among authors: aoyagi scharber m. J Med Chem. 2016 Jan 14;59(1):335-57. doi: 10.1021/acs.jmedchem.5b01498. Epub 2015 Dec 23. J Med Chem. 2016. PMID: 26652717
Trapping Poly(ADP-Ribose) Polymerase.
Shen Y, Aoyagi-Scharber M, Wang B. Shen Y, et al. Among authors: aoyagi scharber m. J Pharmacol Exp Ther. 2015 Jun;353(3):446-57. doi: 10.1124/jpet.114.222448. Epub 2015 Mar 10. J Pharmacol Exp Ther. 2015. PMID: 25758918
Neutral endopeptidase-resistant C-type natriuretic peptide variant represents a new therapeutic approach for treatment of fibroblast growth factor receptor 3-related dwarfism.
Wendt DJ, Dvorak-Ewell M, Bullens S, Lorget F, Bell SM, Peng J, Castillo S, Aoyagi-Scharber M, O'Neill CA, Krejci P, Wilcox WR, Rimoin DL, Bunting S. Wendt DJ, et al. Among authors: aoyagi scharber m. J Pharmacol Exp Ther. 2015 Apr;353(1):132-49. doi: 10.1124/jpet.114.218560. Epub 2015 Feb 3. J Pharmacol Exp Ther. 2015. PMID: 25650377
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: aoyagi scharber m. 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.
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