Design of a versatile nanoparticle-based delivery system for intracellular and in vivo-targeted protein replacement therapy

Autor: Galliani, Marianna

Protein replacement therapy is to-date one of the main clinical approaches to treat a wide number of diseases that are correlated to a protein dysfunction or absence. However, the systemic administration of proteins rarely translates into a successfu

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https://hdl.handle.net/11384/85931

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

Autor: Ilaria Tonazzini, Lucia Angella, Giovanni Signore, Marco Cecchini, Ambra Del Grosso, Melissa Santi, Gabriele Parlanti, Marianna Galliani

Publikováno v: Science Advances
Science Advances 5 (2019). doi:10.1126/sciadv.aax7462
info:cnr-pdr/source/autori:Grosso A.D.; Galliani M.; Angella L.; Santi M.; Tonazzini I.; Parlanti G.; Signore G.; Cecchini M./titolo:Brain-targeted enzyme-loaded nanoparticles: A breach through the blood-brain barrier for enzyme replacement therapy in Krabbe disease/doi:10.1126%2Fsciadv.aax7462/rivista:Science Advances/anno:2019/pagina_da:/pagina_a:/intervallo_pagine:/volume:5

GALC-loaded nanoparticles can reinstate enzymatic activity in the brain of the mouse model of Krabbe disease.
Lysosomal storage disorders (LSDs) result from an enzyme deficiency within lysosomes. The systemic administration of the missing enzyme

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::fd6be6d22f15e1e87f0f3804d48216db
http://hdl.handle.net/11568/1139854