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
2008
Correction of a splice-site mutation in the beta-globin gene stimulated by triplex-forming peptide nucleic acids
Chin JY, Kuan JY, Lonkar PS, Krause DS, Seidman MM, Peterson KR, Nielsen PE, Kole R, Glazer PM. Correction of a splice-site mutation in the beta-globin gene stimulated by triplex-forming peptide nucleic acids. Proceedings Of The National Academy Of Sciences Of The United States Of America 2008, 105: 13514-13519. PMID: 18757759, PMCID: PMC2533221, DOI: 10.1073/pnas.0711793105.Peer-Reviewed Original ResearchConceptsBeta-globin geneEndogenous beta-globin locusSplice site mutationHuman cellsPrimary hematopoietic progenitor cellsBeta-globin locusAltered helical structureHuman beta-globin geneSingle base pair changeSingle base-pair modificationTriplex-forming peptide nucleic acidsDonor DNA moleculesBase pair changesCell cycle stageDisease-related genesDonor DNA fragmentsNucleic acidsProper splicingSite-specific bindingSite-specific modificationMammalian cellsHematopoietic progenitor cellsDNA repairSecond intronGene locus