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Items: 10


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.

Drug Deliv Transl Res. 2020 Jan 15. doi: 10.1007/s13346-019-00683-6. [Epub ahead of print]


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.

Mol Ther Methods Clin Dev. 2019 May 24;14:56-63. doi: 10.1016/j.omtm.2019.05.008. eCollection 2019 Sep 13.


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.

PLoS One. 2019 Jan 18;14(1):e0207836. doi: 10.1371/journal.pone.0207836. eCollection 2019.


Utilizing ExAC to assess the hidden contribution of variants of unknown significance to Sanfilippo Type B incidence.

Clark WT, Yu GK, Aoyagi-Scharber M, LeBowitz JH.

PLoS One. 2018 Jul 6;13(7):e0200008. doi: 10.1371/journal.pone.0200008. eCollection 2018.


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.

Mol Ther Methods Clin Dev. 2017 Jun 6;6:43-53. doi: 10.1016/j.omtm.2017.05.009. eCollection 2017 Sep 15.


Trapping Poly(ADP-Ribose) Polymerase.

Shen Y, Aoyagi-Scharber M, Wang B.

J Pharmacol Exp Ther. 2015 Jun;353(3):446-57. doi: 10.1124/jpet.114.222448. Epub 2015 Mar 10.


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.

J Pharmacol Exp Ther. 2015 Apr;353(1):132-49. doi: 10.1124/jpet.114.218560. Epub 2015 Feb 3.


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.

Proc Natl Acad Sci U S A. 2014 Oct 14;111(41):14870-5. doi: 10.1073/pnas.1416660111. Epub 2014 Sep 29.


Structural basis for the inhibition of poly(ADP-ribose) polymerases 1 and 2 by BMN 673, a potent inhibitor derived from dihydropyridophthalazinone.

Aoyagi-Scharber M, Gardberg AS, Yip BK, Wang B, Shen Y, Fitzpatrick PA.

Acta Crystallogr F Struct Biol Commun. 2014 Sep;70(Pt 9):1143-9. doi: 10.1107/S2053230X14015088. Epub 2014 Aug 29.

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