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 pathogenesis.
Gouri Yogalingam, Amanda R. Luu, Heather Prill, Melanie J. Lo, Bryan K. Yip, John Holtzinger, Terri Christianson, Mika Aoyagi-Scharber, Roger Lawrence, Brett E. Crawford, Jonathan H. LeBowitz
This study characterizes two distinct cellular uptake mechanisms for BMN 250 in Sanfilippo B disease, showing CI-MPR-mediated uptake in neurons and astrocytes and receptor-independent uptake in microglia.
PLoS ONE · Jan 18, 2019Peer-reviewedGenetics