title: Cationic vesicles based on non-ionic surfactant and synthetic aminolipids mediate delivery of antisense oligonucleotides into mammalian cells.
abstract: A formulation based on a synthetic aminolipid containing a double-tailed with two saturated alkyl chains along with a non-ionic surfactant polysorbate-80 has been used to form lipoplexes with an antisense oligonucleotide capable of inhibiting the expression of Renilla luciferase mRNA. The resultant lipoplexes were characterized in terms of morphology, Zeta potential, average size, stability and electrophoretic shift assay. The lipoplexes did not show any cytotoxicity in cell culture up to 150 mM concentration. The gene inhibition studies demonstrated that synthetic cationic vesicles based on non-ionic surfactant and the appropriate aminolipid play an important role in enhancing cellular uptake of antisense oligonucleotides obtaining promising results and efficiencies comparable to commercially available cationic lipids in cultured mammalian cells. Based on these results, this amino lipid moiety could be considered as starting point for the synthesis of novel cationic lipids to obtain potential non-viral carriers for antisense and RNA interference therapies.
authors.affiliation: Institute for Advanced Chemistry of Catalonia (IQAC-CSIC), Department of Chemical and Biomolecular Nanotechnology and Networking Research Centre of Bioengineering, Biomaterials and Nanomedicine (CIBER-BBN), Barcelona, Spain.
authors.forename: Santiago
authors.initials: S
authors.lastname: Grijalvo
authors.affiliation: Institute for Advanced Chemistry of Catalonia (IQAC-CSIC), Department of Chemical and Biomolecular Nanotechnology and Networking Research Centre of Bioengineering, Biomaterials and Nanomedicine (CIBER-BBN), Barcelona, Spain.
authors.forename: Adele
authors.initials: A
authors.lastname: Alagia
authors.affiliation: NanoBioCel Group, University of the Basque Country (EHU-UPV), Vitoria and Networking Research Centre of Bioengineering, Biomaterials and Nanomedicine (CIBER-BBN), Vitoria-Gasteiz, Spain.
authors.forename: Gustavo
authors.initials: G
authors.lastname: Puras
authors.affiliation: NanoBioCel Group, University of the Basque Country (EHU-UPV), Vitoria and Networking Research Centre of Bioengineering, Biomaterials and Nanomedicine (CIBER-BBN), Vitoria-Gasteiz, Spain.
authors.forename: Jon
authors.initials: J
authors.lastname: Zárate
authors.affiliation: NanoBioCel Group, University of the Basque Country (EHU-UPV), Vitoria and Networking Research Centre of Bioengineering, Biomaterials and Nanomedicine (CIBER-BBN), Vitoria-Gasteiz, Spain.
authors.forename: Jose Luis
authors.initials: JL
authors.lastname: Pedraz
authors.affiliation: Institute for Advanced Chemistry of Catalonia (IQAC-CSIC), Department of Chemical and Biomolecular Nanotechnology and Networking Research Centre of Bioengineering, Biomaterials and Nanomedicine (CIBER-BBN), Barcelona, Spain. Electronic address: recgma@cid.csic.es.
authors.forename: Ramon
authors.initials: R
authors.lastname: Eritja
mesh_terms: D001692:Biological Transport; D002412:Cations; D002470:Cell Survival / Q000187:drug effects; D004339:Drug Compounding; D015870:Gene Expression; D018014:Gene Transfer Techniques; D006367:HeLa Cells; D006801:Humans; D057927:Hydrophobic and Hydrophilic Interactions; D008081:Liposomes / Q000737:chemistry*; D049410:Luciferases, Renilla / Q000037:antagonists & inhibitors / Q000235:genetics / Q000378:metabolism; D016376:Oligonucleotides, Antisense / Q000138:chemical synthesis / Q000378:metabolism*; D011136:Polysorbates / Q000737:chemistry*; D011437:Propylamines / Q000737:chemistry; D012333:RNA, Messenger / Q000037:antagonists & inhibitors* / Q000235:genetics / Q000378:metabolism; D013501:Surface-Active Agents / Q000737:chemistry*
chemical_list: D002412:Cations; D008081:Liposomes; D016376:Oligonucleotides, Antisense; D011136:Polysorbates; D011437:Propylamines; D012333:RNA, Messenger; D013501:Surface-Active Agents; D049410:Luciferases, Renilla
keywords: Antisense oligonucleotide; Antisense therapy; Cationic lipids; Cationic vesicles; Gene delivery; Non-ionic surfactant vesicles