title: Lysosomal enzymes may cross the blood-brain-barrier by pinocytosis: implications for enzyme replacement therapy.
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.
authors.affiliation: Gene Therapy Center, Hospital de Clinicas de Porto Alegre, RS, Brazil. Electronic address: guibaldo@ig.com.br.
authors.forename: Guilherme
authors.initials: G
authors.lastname: Baldo
authors.affiliation: Gene Therapy Center, Hospital de Clinicas de Porto Alegre, RS, Brazil.
authors.forename: Roberto
authors.initials: R
authors.lastname: Giugliani
authors.affiliation: Gene Therapy Center, Hospital de Clinicas de Porto Alegre, RS, Brazil.
authors.forename: Ursula
authors.initials: U
authors.lastname: Matte
mesh_terms: D000818:Animals; D001812:Blood-Brain Barrier; D004195:Disease Models, Animal; D004705:Endocytosis; D056947:Enzyme Replacement Therapy / Q000379:methods*; D006801:Humans; D016464:Lysosomal Storage Diseases / Q000628:therapy; D008247:Lysosomes / Q000201:enzymology* / Q000378:metabolism; D008962:Models, Theoretical; D010873:Pinocytosis; D011994:Recombinant Proteins / Q000737:chemistry
chemical_list: D011994:Recombinant Proteins