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Updated: May 2, 2026

Setting-up an In Vitro Model of Rat Blood-brain Barrier BBB: A Focus on BBB Impermeability and Receptor-mediated Transport
Published on: June 28, 2014
Lysosomal enzymes may cross the blood-brain-barrier by pinocytosis: implications for enzyme replacement therapy
Guilherme Baldo1, Roberto Giugliani1, Ursula Matte1
1Gene Therapy Center, Hospital de Clinicas de Porto Alegre, RS, Brazil.
Abstract:
Here we hypothesized that the water-soluble lysosomal enzymes may cross the blood-brain-barrier and reach the brain using the mechanism of unspecific fluid-phase endocytosis. We also highlight studies that show that, at higher serum concentrations, a fraction of these proteins can reach the brain after intravenous injection, and we suggest some experiments to study this hypothesis. Finally we discuss the implications of this for treatments such as enzyme replacement of lysosomal storage disorders.
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