title: Bioreducible Poly(Beta-Amino Ester)s for Intracellular Delivery of SiRNA.
abstract: RNA interference (RNAi) is a powerful tool to target and knockdown gene expression in a sequence-specific manner. RNAi can be achieved by the intracellular introduction of SiRNA; however, intracellular SiRNA delivery remains a challenging obstacle. Herein we describe the use of bioreducible nanoparticles formed using poly(beta-amino ester)s (PBAEs) for safe and efficient SiRNA delivery. Methods for polymer synthesis, nanoparticle formation, and SiRNA delivery using these particles are described. A template protocol for nanoparticle screening is presented and can be used to quickly optimize SiRNA delivery for novel applications.
authors.affiliation: Department of Biomedical Engineering, Johns Hopkins University School of Medicine, Baltimore, MD, USA.|Translational Tissue Engineering Center and Institute for Nanobiotechnology, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
authors.forename: Kristen L
authors.initials: KL
authors.lastname: Kozielski
authors.affiliation: Department of Biomedical Engineering, Johns Hopkins University School of Medicine, Baltimore, MD, USA. green@jhu.edu.|Translational Tissue Engineering Center and Institute for Nanobiotechnology, Johns Hopkins University School of Medicine, Baltimore, MD, USA. green@jhu.edu.|Department of Materials Science and Engineering, Johns Hopkins University School of Medicine, Baltimore, MD, USA. green@jhu.edu.|Department of Ophthalmology, Johns Hopkins University School of Medicine, Baltimore, MD, USA. green@jhu.edu.|Department of Neuroscience, Johns Hopkins University School of Medicine, Baltimore, MD, USA. green@jhu.edu.
authors.forename: Jordan J
authors.initials: JJ
authors.lastname: Green
mesh_terms: D045744:Cell Line, Tumor; D055785:Gene Knockdown Techniques; D006801:Humans; D042541:Intracellular Space / Q000378:metabolism*; D010084:Oxidation-Reduction; D011108:Polymers / Q000138:chemical synthesis / Q000737:chemistry* / Q000378:metabolism*; D034741:RNA, Small Interfering / Q000737:chemistry* / Q000235:genetics
chemical_list: D011108:Polymers; D034741:RNA, Small Interfering; C507253:poly(beta-amino ester)
keywords: Gene delivery; Nanoparticle; Polymer; RNA interference; SiRNA; Transfection