title: Comparison of different cationized proteins as biomaterials for nanoparticle-based ocular gene delivery.
abstract: Cationized polymers have been proposed as transfection agents for gene therapy. The present work aims to improve the understanding of the potential use of different cationized proteins (atelocollagen, albumin and gelatin) as nanoparticle components and to investigate the possibility of modulating the physicochemical properties of the resulting nanoparticle carriers by selecting specific protein characteristics in an attempt to improve current ocular gene-delivery approaches. The toxicity profiles, as well as internalization and transfection efficiency, of the developed nanoparticles can be modulated by modifying the molecular weight of the selected protein and the amine used for cationization. The most promising systems are nanoparticles based on intermediate molecular weight gelatin cationized with the endogenous amine spermine, which exhibit an adequate toxicological profile, as well as effective association and protection of pDNA or siRNA molecules, thereby resulting in higher transfection efficiency and gene silencing than the other studied formulations.
authors: Graduate Program in Pharmaceutical Sciences, Faculty of Pharmacy, Federal University of Rio Grande do Sul, 2752 Ipiranga, 90610-000 Porto Alegre, Brazil. Electronic address: iovanni.zorzi@ufrgs.br.
authors: Giovanni K
authors: GK
authors: Zorzi
authors: Department of Pharmacy and Pharmaceutical Technology, Faculty of Pharmacy, University of Santiago de Compostela (USC), Campus Sur, 15782 Santiago de Compostela, Spain. Electronic address: j.e.parraga@gmail.com.
authors: Jenny E
authors: JE
authors: Párraga
authors: Department of Pharmacy and Pharmaceutical Technology, Faculty of Pharmacy, University of Santiago de Compostela (USC), Campus Sur, 15782 Santiago de Compostela, Spain; Molecular Image Group, Health Research Institute, University Clinical Hospital of Santiago de Compostela (IDIS), A Choupana, 15706 Santiago de Compostela, Spain.
authors: Begoña
authors: B
authors: Seijo
authors: Department of Pharmacy and Pharmaceutical Technology, Faculty of Pharmacy, University of Santiago de Compostela (USC), Campus Sur, 15782 Santiago de Compostela, Spain; Molecular Image Group, Health Research Institute, University Clinical Hospital of Santiago de Compostela (IDIS), A Choupana, 15706 Santiago de Compostela, Spain. Electronic address: alejandro.sanchez@usc.es.
authors: Alejandro
authors: A
authors: Sanchez
mesh_terms: D001672:Biocompatible Materials / Q000737:chemistry*; D002412:Cations / Q000737:chemistry*; D002460:Cell Line; D003228:Conjunctiva / Q000166:cytology / Q000187:drug effects; D003315:Cornea / Q000166:cytology / Q000187:drug effects; D004247:DNA / Q000008:administration & dosage; D018014:Gene Transfer Techniques; D006801:Humans; D008970:Molecular Weight; D053758:Nanoparticles / Q000737:chemistry*; D009883:Ophthalmic Solutions; D011506:Proteins / Q000737:chemistry*; D034741:RNA, Small Interfering / Q000008:administration & dosage
chemical_list: D001672:Biocompatible Materials; D002412:Cations; D009883:Ophthalmic Solutions; D011506:Proteins; D034741:RNA, Small Interfering; D004247:DNA
keywords: Cationized proteins; Gene therapy; Nanoparticles; pDNA; siRNA