title: Human serum albumin-based nanoparticle-mediated in vitro gene delivery.
abstract: The genetic treatment of neurodegenerative diseases still remains a challenging task since many approaches fail to deliver the therapeutic material in relevant concentrations into the brain. As viral vectors comprise the risk of immune and inflammatory responses, human serum albumin (HSA) nanoparticles were found to represent a safer and more convenient alternative. Their ability to cross the blood-brain barrier (BBB) and deliver drugs into the brain in order to enhance gene-based therapy has been previously demonstrated. The present study deals with the development of pGL3-PEI-coated HSA nanoparticles and subsequent in vitro testing in cerebellar granular and HeLa cells. The luciferase control vector pGL3 was chosen as reporter plasmid encoding for the firefly luciferase protein, linear polyethylenimine (22 kDa) as endosomolytic agent for enhancing the cells' transfection. Studies on particle characteristics, their cellular uptake into aforementioned cell lines and on subcellular localisation, and transfection efficiency in the cerebellar cells proved the feasibility of nanoparticle-based gene delivery.
authors.affiliation: Center for Membrane Proteomics, Goethe University, Frankfurt am Main, Germany.
authors.forename: Monica
authors.initials: M
authors.lastname: Langiu
authors.affiliation: Center for Membrane Proteomics, Goethe University, Frankfurt am Main, Germany; Institute of Pharmaceutical Technology, Goethe University, Frankfurt am Main, Germany.
authors.forename: Miriam
authors.initials: M
authors.lastname: Dadparvar
authors.affiliation: Institute of Pharmaceutical Technology, Goethe University, Frankfurt am Main, Germany.
authors.forename: Jörg
authors.initials: J
authors.lastname: Kreuter
authors.affiliation: Center for Membrane Proteomics, Goethe University, Frankfurt am Main, Germany.
authors.forename: Mika O
authors.initials: MO
authors.lastname: Ruonala
mesh_terms: D000818:Animals; D002531:Cerebellum / Q000166:cytology / Q000378:metabolism; D004247:DNA / Q000737:chemistry / Q000235:genetics; D004337:Drug Carriers / Q000737:chemistry* / Q000378:metabolism; D004705:Endocytosis; D006367:HeLa Cells; D006801:Humans; D051379:Mice; D053758:Nanoparticles / Q000737:chemistry*; D009474:Neurons / Q000378:metabolism; D010316:Particle Size; D010957:Plasmids / Q000235:genetics; D011094:Polyethyleneimine / Q000737:chemistry*; D012709:Serum Albumin / Q000737:chemistry*; D014162:Transfection
chemical_list: D004337:Drug Carriers; D012709:Serum Albumin; D011094:Polyethyleneimine; D004247:DNA