2016
In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery
Bahal R, Ali McNeer N, Quijano E, Liu Y, Sulkowski P, Turchick A, Lu YC, Bhunia DC, Manna A, Greiner DL, Brehm MA, Cheng CJ, López-Giráldez F, Ricciardi A, Beloor J, Krause DS, Kumar P, Gallagher PG, Braddock DT, Mark Saltzman W, Ly DH, Glazer PM. In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery. Nature Communications 2016, 7: 13304. PMID: 27782131, PMCID: PMC5095181, DOI: 10.1038/ncomms13304.Peer-Reviewed Original ResearchConceptsNanoparticle deliveryGene correctionReversal of splenomegalyPeptide nucleic acidLow off-target effectsVivo correctionGenome editingOff-target effectsGene editingHaematopoietic stem cellsNucleic acidsDonor DNAStem cellsΓPNAΒ-thalassaemiaNanoparticlesDeliveryEditingSCF treatmentTriplex formation
2014
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 ResearchConceptsGene 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