2022
Nanoparticle‐mediated genome editing in single‐cell embryos via peptide nucleic acids
Putman R, Ricciardi A, Carufe K, Quijano E, Bahal R, Glazer P, Saltzman W. Nanoparticle‐mediated genome editing in single‐cell embryos via peptide nucleic acids. Bioengineering & Translational Medicine 2022, 8: e10458. PMID: 37206203, PMCID: PMC10189434, DOI: 10.1002/btm2.10458.Peer-Reviewed Original ResearchSingle-cell embryosPeptide nucleic acidGene editingNucleic acidsNanoparticlesGross developmental abnormalitiesGenome editingNormal physiological developmentOff-target effectsDonor DNAGenetic diseasesConcept workEmbryosGenomic effectsDevelopmental abnormalitiesNormal growthEmbryogenesisPhysiological developmentEditingUnderlying mutationPreimplantation genetic diagnosisDisease pathogenesisGenetic diagnosisNormal morphologyAcid
2021
The NIH Somatic Cell Genome Editing program
Saha K, Sontheimer EJ, Brooks PJ, Dwinell MR, Gersbach CA, Liu DR, Murray SA, Tsai SQ, Wilson RC, Anderson DG, Asokan A, Banfield JF, Bankiewicz KS, Bao G, Bulte JWM, Bursac N, Campbell JM, Carlson DF, Chaikof EL, Chen ZY, Cheng RH, Clark KJ, Curiel DT, Dahlman JE, Deverman BE, Dickinson ME, Doudna JA, Ekker SC, Emborg ME, Feng G, Freedman BS, Gamm DM, Gao G, Ghiran IC, Glazer PM, Gong S, Heaney JD, Hennebold JD, Hinson JT, Khvorova A, Kiani S, Lagor WR, Lam KS, Leong KW, Levine JE, Lewis JA, Lutz CM, Ly DH, Maragh S, McCray PB, McDevitt TC, Mirochnitchenko O, Morizane R, Murthy N, Prather RS, Ronald JA, Roy S, Roy S, Sabbisetti V, Saltzman WM, Santangelo PJ, Segal DJ, Shimoyama M, Skala MC, Tarantal AF, Tilton JC, Truskey GA, Vandsburger M, Watts JK, Wells KD, Wolfe SA, Xu Q, Xue W, Yi G, Zhou J. The NIH Somatic Cell Genome Editing program. Nature 2021, 592: 195-204. PMID: 33828315, PMCID: PMC8026397, DOI: 10.1038/s41586-021-03191-1.Peer-Reviewed Original ResearchConceptsDownstream functional consequencesGenome modificationHuman genomeGenome editingGenome editorsSomatic cellsHuman cellsFunctional consequencesBiomedical research communityGenomeLarge animalsBiological systemsCellsHuman healthHuman biological systemsEditingVivoAnimal modelsNew therapiesNew opportunitiesWide rangeConsortium
2018
Debugging the genetic code: non-viral in vivo delivery of therapeutic genome editing technologies
Piotrowski-Daspit AS, Glazer P, Saltzman WM. Debugging the genetic code: non-viral in vivo delivery of therapeutic genome editing technologies. Current Opinion In Biomedical Engineering 2018, 7: 24-32. PMID: 30984891, PMCID: PMC6456264, DOI: 10.1016/j.cobme.2018.08.002.Peer-Reviewed Original ResearchGenome editingNon-viral delivery methodsCRISPR/Cas9 systemGenome engineering technologiesGenome editing technologyTherapeutic genome editingPeptide nucleic acidSpecific cell typesGenetic codeVivo deliveryCas9 systemEditing technologyEfficient deliveryGenomic mutationsCell typesPolymeric vehiclesFuture outlookDisease phenotypePrecise technologyEngineering technologyDelivery methodsNucleic acidsCell culturesEditingHereditary diseaseIn 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
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 formationPrecise 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