Research ArticleBLOOD-BRAIN BARRIER

Brain delivery and activity of a lysosomal enzyme using a blood-brain barrier transport vehicle in mice

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Science Translational Medicine  27 May 2020:
Vol. 12, Issue 545, eaay1163
DOI: 10.1126/scitranslmed.aay1163

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Transport vehicle for CNS therapeutics

Delivering biotherapeutics to the brain is complicated by the presence of the blood-brain barrier (BBB). Kariolis et al. and Ullman et al. developed a transport vehicle (TV) consisting of an Fc fragment engineered to bind to the transferrin receptor, a protein highly expressed at the BBB. Utilizing the TV in the form of an antibody directed against β-secretase enhanced brain delivery and effects in both mice and monkeys following systemic administration. Systemic delivery of a TV fusion with the iduronate 2-sulfatase enzyme was effective at reducing peripheral and central pathologies in a mouse model of mucopolysaccharidosis type II. The TV platform approach potentially offers a treatment for neurological disorders.

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