Published in

American Association for the Advancement of Science, Science Advances, 11(5), 2019

DOI: 10.1126/sciadv.aax7462

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Brain-targeted enzyme-loaded nanoparticles: A breach through the blood-brain barrier for enzyme replacement therapy in Krabbe disease

This paper is made freely available by the publisher.
This paper is made freely available by the publisher.

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Abstract

GALC-loaded nanoparticles can reinstate enzymatic activity in the brain of the mouse model of Krabbe disease.