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, Bing, Chu, Daniel, Feng, Ying, Shen, Yuqiao, Aoyagi-Scharber, Mika, Post, Leonard E
Published in Journal of medicinal chemistry (14.01.2016)
Published in Journal of medicinal chemistry (14.01.2016)
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Journal Article
Neutral endopeptidase-resistant C-type natriuretic peptide variant represents a new therapeutic approach for treatment of fibroblast growth factor receptor 3-related dwarfism
Wendt, Daniel J, Dvorak-Ewell, Melita, Bullens, Sherry, Lorget, Florence, Bell, Sean M, Peng, Jeff, Castillo, Sianna, Aoyagi-Scharber, Mika, O'Neill, Charles A, Krejci, Pavel, Wilcox, William R, Rimoin, David L, Bunting, Stuart
Published in The Journal of pharmacology and experimental therapeutics (01.04.2015)
Published in The Journal of pharmacology and experimental therapeutics (01.04.2015)
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Journal Article
Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB
Kan, Shih-hsin, Aoyagi-Scharber, Mika, Le, Steven Q., Vincelette, Jon, Ohmi, Kazuhiro, Bullens, Sherry, Wendt, Daniel J., Christiansen, Terri M., Tiger, Pascale M. N., Brown, Julian R., Lawrence, Roger, Yip, Bryan K., Holtzinger, John, Bagri, Anil, Crippen-Harmon, Danielle, Vondrak, Kristen N., Chen, Zhi, Hague, Chuck M., Woloszynek, Josh C., Cheung, Diana S., Webster, Katherine A., Adintori, Evan G., Lo, Melanie J., Wong, Wesley, Fitzpatrick, Paul A., LeBowitz, Jonathan H., Crawford, Brett E., Bunting, Stuart, Dickson, Patricia I., Neufeld, Elizabeth F.
Published in Proceedings of the National Academy of Sciences - PNAS (14.10.2014)
Published in Proceedings of the National Academy of Sciences - PNAS (14.10.2014)
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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, Gouri, Luu, Amanda R, Prill, Heather, Lo, Melanie J, Yip, Bryan, Holtzinger, John, Christianson, Terri, Aoyagi-Scharber, Mika, Lawrence, Roger, Crawford, Brett E, LeBowitz, Jonathan H
Published in PloS one (18.01.2019)
Published in PloS one (18.01.2019)
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Structural basis for the inhibition of poly(ADP-ribose) polymerases 1 and 2 by BMN 673, a potent inhibitor derived from dihydropyridophthalazinone
Aoyagi-Scharber, Mika, Gardberg, Anna S., Yip, Bryan K., Wang, Bing, Shen, Yuqiao, Fitzpatrick, Paul A.
Published in Acta crystallographica. Section F, Structural biology communications (01.09.2014)
Published in Acta crystallographica. Section F, Structural biology communications (01.09.2014)
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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, 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
Published in Drug delivery and translational research (01.04.2020)
Published in Drug delivery and translational research (01.04.2020)
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Journal Article
Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice
Aoyagi-Scharber, Mika, Crippen-Harmon, Danielle, Lawrence, Roger, Vincelette, Jon, Yogalingam, Gouri, Prill, Heather, Yip, Bryan K, Baridon, Brian, Vitelli, Catherine, Lee, Amanda, Gorostiza, Olivia, Adintori, Evan G, Minto, Wesley C, Van Vleet, Jeremy L, Yates, Bridget, Rigney, Sara, Christianson, Terri M, Tiger, Pascale M N, Lo, Melanie J, Holtzinger, John, Fitzpatrick, Paul A, LeBowitz, Jonathan H, Bullens, Sherry, Crawford, Brett E, Bunting, Stuart
Published in Molecular therapy. Methods & clinical development (15.09.2017)
Published in Molecular therapy. Methods & clinical development (15.09.2017)
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Journal Article
Trapping Poly(ADP-Ribose) Polymerase
Shen, Yuqiao, Aoyagi-Scharber, Mika, Wang, Bing
Published in The Journal of pharmacology and experimental therapeutics (01.06.2015)
Published in The Journal of pharmacology and experimental therapeutics (01.06.2015)
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Journal Article
Discovery and Characterization of (8 S ,9 R )-5-Fluoro-8-(4-fluorophenyl)-9-(1-methyl-1 H -1,2,4-triazol-5-yl)-2,7,8,9-tetrahydro-3 H -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, Bing, Chu, Daniel, Feng, Ying, Shen, Yuqiao, Aoyagi-Scharber, Mika, Post, Leonard E.
Published in Journal of medicinal chemistry (14.01.2016)
Published in Journal of medicinal chemistry (14.01.2016)
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Journal Article
LATE BREAKING NEWS E-POSTER PRESENTATION: LBP 7 Characterization of Novel Exon-51 Skipping Oligonucleotides that Safely Rescues Dystrophin Expression in a Severe Model of Duchenne Muscular Dystrophy
ONeill, Charles, Hedlund, Maria, Chandra, Sundeep, Hock, M Benjamin, Melton, Andrew, Oppeneer, Todd, Cherala, Ganesh, Aoyagi-Scharber, Mika, Frazer-Abel, Ashley, Datson, Nicole, van Deutekom, Judith, Poulivali, Jukka, Fang, Xiefan, Fong, Sylvia, Jacoby, David, Bhagwat, Shripad
Published in Neuromuscular disorders : NMD (01.10.2020)
Published in Neuromuscular disorders : NMD (01.10.2020)
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Journal Article
LATE BREAKING NEWS E-POSTER PRESENTATION
ONeill, Charles, Hedlund, Maria, Chandra, Sundeep, Hock, M Benjamin, Melton, Andrew, Oppeneer, Todd, Cherala, Ganesh, Aoyagi-Scharber, Mika, Frazer-Abel, Ashley, Datson, Nicole, van Deutekom, Judith, Poulivali, Jukka, Fang, Xiefan, Fong, Sylvia, Jacoby, David, Bhagwat, Shripad
Published in Neuromuscular disorders : NMD (01.10.2020)
Published in Neuromuscular disorders : NMD (01.10.2020)
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Journal Article
Utilizing activity assays and population-wide allele frequencies to assess the contribution of novel mutations in NAGLU to MPS IIIB incidence
LeBowitz, Jonathan H., Clark, Wyatt T., Karen Yu, G., Aoyagi-Scharber, Mika
Published in Molecular genetics and metabolism (01.02.2016)
Published in Molecular genetics and metabolism (01.02.2016)
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Journal Article
Translational dose-response and frequency scaling for BMN 250 administered via the intracerebroventricular route: Predicting a clinically effective dosing regimen from animal models of disease for the treatment of Sanfilippo syndrome type B
Grover, Anita, McCullagh, Emma, Aoyagi-Scharber, Mika, Maricich, Stephen M., Wait, Jill C.M., Melton, Andrew, Zhou, Huiyu, Lawrence, Roger, Prill, Heather, Crawford, Brett, Henshaw, Joshua
Published in Molecular genetics and metabolism (01.02.2018)
Published in Molecular genetics and metabolism (01.02.2018)
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Journal Article
Differential Uptake of NAGLU-IGF2 and Unmodified NAGLU in Cellular Models of Sanfilippo Syndrome Type B
Prill, Heather, Luu, Amanda, Yip, Bryan, Holtzinger, John, Lo, Melanie J., Christianson, Terri M., Yogalingam, Gouri, Aoyagi-Scharber, Mika, LeBowitz, Jonathan H., Crawford, Brett E., Lawrence, Roger
Published in Molecular therapy. Methods & clinical development (13.09.2019)
Published in Molecular therapy. Methods & clinical development (13.09.2019)
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Journal Article
Glycosylation independent lysosomal targeting of alpha-n-acetylglucosaminidase confers highly efficient enzyme uptake into critical cellular targets of disease pathogenesis in mucopolysaccharidosis type IIIB
Yogalingam, Gouri, Prill, Heather, Lee, Amanda, Christianson, Terri, Tiger, Pascale M.N., Yip, Bryan K., Lo, Melanie J., Holtzinger, John, Berguig, Geoffrey, Aoyagi-Scharber, Mika, Fitzpatrick, Paul, Lawrence, Roger, Crawford, Brett, LeBowitz, Jonathan H.
Published in Molecular genetics and metabolism (01.02.2016)
Published in Molecular genetics and metabolism (01.02.2016)
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Journal Article
Intracerebroventricular enzyme replacement therapy with glycosylation-independent lysosomal targeted NAGLU leads to widespread enzymatic activity, reduction of lysosomal storage and of secondary defects in brain of mice with Sanfilippo syndrome type B
Kan, Shih-hsin, Le, Steven, Vincelette, Jon, Bullens, Sherron, Brown, Jillian, Ohmi, Kazuhiro, Lotshaw, Ethan, Aoyagi-Scharber, Mika, Crawford, Brett E., Bunting, Stuart, Neufeld, Elizabeth, Dickson, Patricia I.
Published in Molecular genetics and metabolism (01.02.2014)
Published in Molecular genetics and metabolism (01.02.2014)
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Journal Article
Time- and dose-dependent normalization of pathological lysosomal storage and biochemistry in the mucopolysaccharidosis ΙΙΙΒ (MPS ΙΙΙΒ, Sanfilippo syndrome type Β) mouse model by intracerebroventricular enzyme replacement therapy with ΒΜΝ 250, a ΝAGLU-ΙGF2 fusion pro
Aoyagi-Scharber, Mika, Vincelette, Jon, Lawrence, Roger, Crippen-Harmon, Danielle, Yip, Bryan K., Baridon, Brian, Prill, Heather, Minto, Wesley C., Van Vleet, Jeremy L., Vitelli, Catherine, Adintori, Evan G., Christianson, Terri, Tiger, Pascale M.N., Lo, Melanie J., Holtzinger, John, Chen, Eric, Webster, Katherine A., Brown, Jillian R., Fitzpatrick, Paul A., LeBowitz, Jonathan H., Bagri, Anil, Bullens, Sherry, Crawford, Brett E., Bunting, Stuart
Published in Molecular genetics and metabolism (01.02.2016)
Published in Molecular genetics and metabolism (01.02.2016)
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Journal Article
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
Bagri, Anil, Harmon, Danielle, Vincelette, Jon, Vitelli, Catherine, Minto, Wesley, Yates, Bridget, Rigney, Sara, Santiago, Pam, Baridon, Brian, Xie, Lin, Gorostiza, Olivia, Christianson, Terri, Tiger, Pascale M.N., Yip, Bryan K., Lo, Melanie J., Holtzinger, John, Chen, Eric, Aoyagi-Scharber, Mika, Fitzpatrick, Paul, LeBowitz, Jonathan H., Bullens, Sherry, Bunting, Stuart
Published in Molecular genetics and metabolism (01.02.2016)
Published in Molecular genetics and metabolism (01.02.2016)
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