Research ArticleBLOOD-BRAIN BARRIER

Brain delivery of therapeutic proteins using an Fc fragment blood-brain barrier transport vehicle in mice and monkeys

See allHide authors and affiliations

Science Translational Medicine  27 May 2020:
Vol. 12, Issue 545, eaay1359
DOI: 10.1126/scitranslmed.aay1359

You are currently viewing the editor's summary.

View Full Text

Log in to view the full text

Log in through your institution

Log in through your institution

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 beta-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.

View Full Text

Stay Connected to Science Translational Medicine