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...

by Gouri Yogalingam, Amanda R Luu, Heather Prill, Melanie J Lo, Bryan Yip, John Holtzinger, Terri Christianson, Mika Aoyagi-Scharber, Roger Lawrence, Brett E Crawford, Jonathan H LeBowitz
Published 2019-01-01

Characterization of glycan substrates accumulating in GM1 Gangliosidosis

by Roger Lawrence, Jeremy L. Van Vleet, Linley Mangini, Adam Harris, Nathan Martin, Wyatt Clark, Sanjay Chandriani, Jonathan H. LeBowitz, Roberto Giugliani, Alessandra d'Azzo, Gouri Yogalingam, Brett E. Crawford
Published 2019-12-01

Identifying therapeutic drug targets using bidirectional effect genes

by Karol Estrada, Steven Froelich, Arthur Wuster, Christopher R. Bauer, Teague Sterling, Wyatt T. Clark, Yuanbin Ru, Marena Trinidad, Hong Phuc Nguyen, Amanda R. Luu, Daniel J. Wendt, Gouri Yogalingam, Guoying Karen Yu, Jonathan H. LeBowitz, Lon R. Cardon
Published 2021-04-01

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

by Mika Aoyagi-Scharber, Danielle Crippen-Harmon, Roger Lawrence, Jon Vincelette, Gouri Yogalingam, Heather Prill, Bryan K. Yip, Brian Baridon, Catherine Vitelli, Amanda Lee, Olivia Gorostiza, Evan G. Adintori, Wesley C. Minto, Jeremy L. Van Vleet, Bridget Yates, Sara Rigney, Terri M. Christianson, Pascale M.N. Tiger, Melanie J. Lo, John Holtzinger, Paul A. Fitzpatrick, Jonathan H. LeBowitz, Sherry Bullens, Brett E. Crawford, Stuart Bunting
Published 2017-09-01