Single-stranded γPNAs for in vivo site-specific genome editing via Watson-Crick recognition.
Bahal R, Quijano E, McNeer NA, Liu Y, Bhunia DC, Lopez-Giraldez F, Fields RJ, Saltzman WM, Ly DH, Glazer PM. Single-stranded γPNAs for in vivo site-specific genome editing via Watson-Crick recognition. Current Gene Therapy 2014, 14: 331-42. PMID: 25174576, PMCID: PMC4333085, DOI: 10.2174/1566523214666140825154158.Peer-Reviewed Original ResearchMeSH KeywordsAnimalsBeta-GlobinsBone MarrowDNAGene TargetingGreen Fluorescent ProteinsHigh-Throughput Nucleotide SequencingHumansLactic AcidMiceMice, TransgenicNanoparticlesPeptide Nucleic AcidsPolyglycolic AcidPolylactic Acid-Polyglycolic Acid CopolymerRNA EditingTissue DonorsConceptsGene editingSite-specific genome editingEfficient intracellular deliverySite-specific formationIntracellular deliveryGamma PNAsGenome editingAltered helical structureGenomic DNANext generationDonor DNAΓPNANovel strategyMouse bone marrow cellsNanoparticlesDNA repairEditingMouse bone marrowHomopurine sitesSequence restrictionDetectable toxicityBone marrow cellsDNAHereinHelical structure