NeuroscienceThe Journal of Experimental MedicinePeer-reviewed
Molecular architecture determines brain delivery of a transferrin-receptor targeted lysosomal enzyme
Annie Arguello, Cathal S. Mahon, Meredith Calvert…·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.
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