A modular RNA delivery system comprising spherical nucleic acids built on endosome-escaping polymeric nanoparticles
Antonio Garcia-Guerra, Ruth Ellerington, Jens Gaitzsch, Jonathan Bath, Mahnseok Kye, Miguel A. Varela, Giuseppe Battaglia, Matthew J. A. Wood, Raquel Manzano, Carlo Rinaldi, Andrew J. Turberfield
Abstract
, a genetic target with therapeutic potential for amyotrophic lateral sclerosis, to motor neuron-like cells. By attaching a neuron-specific targeting peptide to the PSNA corona, effective knock-down is achieved at doses of 2 particles per cell.
Topics & Concepts
Nucleic acidEndosomeOligonucleotideRNANanoparticlePolymerConjugated systemNanotechnologyChemistryConjugateDNAMaterials scienceBiochemistryOrganic chemistryGeneCellMathematicsMathematical analysisRNA Interference and Gene DeliveryAdvanced biosensing and bioanalysis techniquesNanopore and Nanochannel Transport Studies