2018
In utero nanoparticle delivery for site-specific genome editing
Ricciardi AS, Bahal R, Farrelly JS, Quijano E, Bianchi AH, Luks VL, Putman R, López-Giráldez F, Coşkun S, Song E, Liu Y, Hsieh WC, Ly DH, Stitelman DH, Glazer PM, Saltzman WM. In utero nanoparticle delivery for site-specific genome editing. Nature Communications 2018, 9: 2481. PMID: 29946143, PMCID: PMC6018676, DOI: 10.1038/s41467-018-04894-2.Peer-Reviewed Original ResearchConceptsSite-specific genome editingReversal of splenomegalyPeptide nucleic acidIntra-amniotic administrationBlood hemoglobin levelsMonogenic disordersNanoparticle deliveryPolymeric nanoparticlesPostnatal elevationGestational ageHemoglobin levelsImproved survivalPediatric morbidityDisease improvementHuman β-thalassemiaReticulocyte countNormal organ developmentMouse modelNormal rangeEarly interventionGenome editingOff-target mutationsPostnatal growthGene editingVersatile method
2016
Precise Genome Modification Using Triplex Forming Oligonucleotides and Peptide Nucleic Acids
Bahal R, Gupta A, Glazer P. Precise Genome Modification Using Triplex Forming Oligonucleotides and Peptide Nucleic Acids. Advances In Experimental Medicine And Biology 2016, 93-110. DOI: 10.1007/978-1-4939-3509-3_6.Peer-Reviewed Original ResearchHomologous recombinationSite-specific genome editingPrecise genome modificationGenomic DNA sequencesSpecific genome editingTriplex technologySingle base pair mutationTriplex forming oligonucleotideBase pair mutationGenetic disordersGenome modificationNucleic acidsDefective protein synthesisGenome editingDNA sequencesDifferent genesPair mutationsProtein synthesisMutation sitesGenesSpecific sitesGene replacement therapyExpressionOligonucleotideMutagenesis