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
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