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

por Clark, Wyatt T., Yu, G. Karen, Aoyagi-Scharber, Mika, LeBowitz, Jonathan H.
Publicado 2018

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

por Aoyagi-Scharber, Mika, Gardberg, Anna S., Yip, Bryan K., Wang, Bing, Shen, Yuqiao, Fitzpatrick, Paul A.
Publicado 2014

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 pat...

por 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.
Publicado 2019

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

por 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
Publicado 2019

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

por 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
Publicado 2017

Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatm...

por 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
Publicado 2020