Prednisolone Does Not Regulate Factor VIII Expression in Mice Receiving AAV5-hFVIII-SQ: Valoctocogene Roxaparvovec

by Lening Zhang, Britta Handyside, Ryan Murphy, Choong-Ryoul Sihn, Lin Xie, Catherine Vitelli, Danielle Harmon, Sílvia Sisó, Su Liu, Sherry Bullens, Stuart Bunting, Sylvia Fong
Published 2020-06-01

Induction of ER Stress by an AAV5 BDD FVIII Construct Is Dependent on the Strength of the Hepatic-Specific Promoter

by Sylvia Fong, Britta Handyside, Choong-Ryoul Sihn, Su Liu, Lening Zhang, Lin Xie, Ryan Murphy, Nicole Galicia, Bridget Yates, Wesley C. Minto, Catherine Vitelli, Danielle Harmon, Yuanbin Ru, Guoying Karen Yu, Claudia Escher, Jakob Vowinckel, Jill Woloszynek, Hassib Akeefe, Rajeev Mahimkar, Sherry Bullens, Stuart Bunting
Published 2020-09-01

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