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

Intracerebroventricular enzyme replacement therapy with β-galactosidase reverses brain pathologies due to GM1 gangliosidosis in mice

por Chen, Joseph C., Luu, Amanda R., Wise, Nathan, Angelis, Rolando De, Agrawal, Vishal, Mangini, Linley, Vincelette, Jon, Handyside, Britta, Sterling, Harry, Lo, Melanie J., Wong, Hio, Galicia, Nicole, Pacheco, Glenn, Van Vleet, Jeremy, Giaramita, Alexander, Fong, Sylvia, Roy, Sushmita M., Hague, Chuck, Lawrence, Roger, Bullens, Sherry, Christianson, Terri M., d'Azzo, Alessandra, Crawford, Brett E., Bunting, Stuart, LeBowitz, Jonathan H., Yogalingam, Gouri
Publicado 2020

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

Intracerebroventricular dosing of N-sulfoglucosamine sulfohydrolase in mucopolysaccharidosis IIIA mice reduces markers of brain lysosomal dysfunction

por Magat, Jenna, Jones, Samantha, Baridon, Brian, Agrawal, Vishal, Wong, Hio, Giaramita, Alexander, Mangini, Linley, Handyside, Britta, Vitelli, Catherine, Parker, Monica, Yeung, Natasha, Zhou, Yu, Pungor, Erno, Slabodkin, Ilya, Gorostiza, Olivia, Aguilera, Allora, Lo, Melanie J., Alcozie, Saida, Christianson, Terri M., Tiger, Pascale M.N., Vincelette, Jon, Fong, Sylvia, Gil, Geuncheol, Hague, Chuck, Lawrence, Roger, Wendt, Daniel J., Lebowitz, Jonathan H., Bunting, Stuart, Bullens, Sherry, Crawford, Brett E., Roy, Sushmita M., Woloszynek, Josh C.
Publicado 2022