M. Aoyagi-Scharber

发表

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.

B. Wang, L. Post, Ying Feng, 2016, Journal of medicinal chemistry.

Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB

P. Fitzpatrick, S. Bunting, A. Bagri, 2014, Proceedings of the National Academy of Sciences.

Differential Uptake of NAGLU-IGF2 and Unmodified NAGLU in Cellular Models of Sanfilippo Syndrome Type B

J. LeBowitz, R. Lawrence, Bryan K. Yip, 2019, Molecular therapy. Methods & clinical development.

Neutral Endopeptidase-Resistant C-Type Natriuretic Peptide Variant Represents a New Therapeutic Approach for Treatment of Fibroblast Growth Factor Receptor 3–Related Dwarfism

D. Rimoin, W. Wilcox, S. Bunting, 2015, The Journal of Pharmacology and Experimental Therapeutics.

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

W. Clark, J. LeBowitz, M. Aoyagi-Scharber, 2018, bioRxiv.

Trapping Poly(ADP-Ribose) Polymerase

B. Wang, Bing Wang, Mika Aoyagi-Scharber, 2015, The Journal of Pharmacology and Experimental Therapeutics.

Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice

P. Fitzpatrick, S. Bunting, Catherine Vitelli, 2017, Molecular therapy. Methods & clinical development.

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

J. LeBowitz, R. Lawrence, Bryan K. Yip, 2018, bioRxiv.

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

A. Shaywitz, P. Fitzpatrick, S. Bunting, 2020, Drug Delivery and Translational Research.

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

A. Shaywitz, P. Fitzpatrick, S. Bunting, 2020, Drug Delivery and Translational Research.

Histologic characterization of the progression of central nervous system pathology in the mucopolysaccharidosis IIIB (MPS IIIB, Sanfilippo syndrome type B) mouse model and bio-distribution and efficacy of the intracerebroventricular enzyme replacement therapy, BMN 250

P. Fitzpatrick, S. Bunting, A. Bagri, 2016 .

Engineering of a recombinant NAGLU fusion protein with insulin-like growth factor 2 leads to improved cellular uptake via a glycosylation-independent lysosomal targeting pathway

P. Fitzpatrick, P. Dickson, Bryan K. Yip, 2014 .

Structural Biology Communications Structural Basis for the Inhibition of Poly(adp- Ribose) Polymerases 1 and 2 by Bmn 673, a Potent Inhibitor Derived from Dihydropyridophthalazinone

P. Fitzpatrick, B. Wang, Anna S. Gardberg, 2022 .

Neutral Endopeptidase-Resistant C-Type Natriuretic Peptide Variant Represents a New Therapeutic Approach for Treatment of Fibroblast Growth Factor Receptor 3–Related Dwarfism

D. Rimoin, W. Wilcox, S. Bunting, 2015, The Journal of Pharmacology and Experimental Therapeutics.

Trapping Poly(ADP-Ribose) Polymerase

B. Wang, Yuqiao Shen, M. Aoyagi-Scharber, 2015, The Journal of Pharmacology and Experimental Therapeutics.