Molecular architecture determines brain delivery of a transferrin-receptor targeted lysosomal enzyme
Annie Arguello, Cathal S. Mahon, Meredith Calvert, Darren Chan, Jason C. Dugas, Michelle E. Pizzo, Elliot R. Thomsen, Roni Chau, Lorna A. Damo, Joseph N. Duque, Timothy Earr, Meng Fang, Tina Giese, Do Jin Kim, Nicholas Liang, , Mihalis S. Kariolis
The paper compares brain delivery and efficacy of two TfR-targeted enzyme fusions in MPS II mice, finding the moderate-affinity ETV:IDS has better brain exposure and substrate reduction than the high-affinity IgG:IDS.
The Journal of Experimental Medicine · Dec 17, 2021Peer-reviewedNeuroscience