Enzyme replacement in a human model of mucopolysaccharidosis IVA in vitro and its biodistribution in the cartilage of wild type mice.

by Melita Dvorak-Ewell, Dan Wendt, Chuck Hague, Terri Christianson, Vish Koppaka, Danielle Crippen, Emil Kakkis, Michel Vellard
Published 2010-01-01

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