2024
The Technical and Ethical Framework of Fetal Therapy: Past and Current Advances
Lynn A, Glazer P, Saltzman W, Stitelman D. The Technical and Ethical Framework of Fetal Therapy: Past and Current Advances. Current Stem Cell Reports 2024, 10: 30-36. DOI: 10.1007/s40778-024-00235-w.Peer-Reviewed Original ResearchFetal drug deliverySuccess of nanomedicineDrug deliveryPharmacological drug treatmentsMinimally invasive strategyFetal therapyFetal applicationsClinical studiesCongenital diseaseInvasive strategyDrug treatmentClinical translationClinical practiceTherapyMedical interventionsDeliveryFetalNanomedicineBirthDiseaseFindingsAClinicians
2022
In vivo correction of cystic fibrosis mediated by PNA nanoparticles
Piotrowski-Daspit AS, Barone C, Lin CY, Deng Y, Wu D, Binns TC, Xu E, Ricciardi AS, Putman R, Garrison A, Nguyen R, Gupta A, Fan R, Glazer PM, Saltzman WM, Egan ME. In vivo correction of cystic fibrosis mediated by PNA nanoparticles. Science Advances 2022, 8: eabo0522. PMID: 36197984, PMCID: PMC9534507, DOI: 10.1126/sciadv.abo0522.Peer-Reviewed Original ResearchCystic fibrosisF508del miceIntravenous deliveryPrimary nasal epithelial cellsMultiple organ dysfunctionNasal epithelial cellsUssing chamber assaysOrgan dysfunctionF508del cystic fibrosisVivo treatmentGI tissuesCF transmembrane conductance regulator (CFTR) geneChamber assaySystemic deliveryEpithelial cellsCF-causing mutationsFibrosisCFTR functionMiceTransmembrane conductance regulator geneTarget effectsAir-liquid interfaceDeliveryPartial gainViable option
2021
Targeting the Hypoxic and Acidic Tumor Microenvironment with pH-Sensitive Peptides
Dharmaratne NU, Kaplan AR, Glazer PM. Targeting the Hypoxic and Acidic Tumor Microenvironment with pH-Sensitive Peptides. Cells 2021, 10: 541. PMID: 33806273, PMCID: PMC8000199, DOI: 10.3390/cells10030541.Peer-Reviewed Original ResearchConceptsNucleic acid analoguesPH-sensitive peptideLow insertion peptideAcidic tumor microenvironmentPoor bioavailabilityInsertion peptideFluorescent dyeHigh toxicitySelective deliveryAcid analoguesSmall molecule inhibitorsNanoparticlesCancer therapeuticsMolecule inhibitorsDyePeptidesReview articleAcidityPHLIPPromising toolBioavailabilityAnaloguesDeliveryHypoxic tumor microenvironment
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
2015
Nanoparticles that deliver triplex-forming peptide nucleic acid molecules correct F508del CFTR in airway epithelium
McNeer NA, Anandalingam K, Fields RJ, Caputo C, Kopic S, Gupta A, Quijano E, Polikoff L, Kong Y, Bahal R, Geibel JP, Glazer PM, Saltzman WM, Egan ME. Nanoparticles that deliver triplex-forming peptide nucleic acid molecules correct F508del CFTR in airway epithelium. Nature Communications 2015, 6: 6952. PMID: 25914116, PMCID: PMC4480796, DOI: 10.1038/ncomms7952.Peer-Reviewed Original ResearchMeSH KeywordsAnimalsCell LineChloridesCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorDNA-Binding ProteinsGenetic TherapyHigh-Throughput Nucleotide SequencingHumansLactic AcidMice, Inbred C57BLNanoparticlesPeptide Nucleic AcidsPolyglycolic AcidPolylactic Acid-Polyglycolic Acid CopolymerPolymersRespiratory MucosaConceptsFacile genome engineeringVivo gene deliveryBiodegradable polymer nanoparticlesTransient gene expressionNanoparticle systemsGene deliveryPolymer nanoparticlesGene correctionGenome engineeringNanoparticlesOff-target effectsPeptide nucleic acidLethal genetic disorderNucleic acidsDonor DNATarget effectsIntranasal deliveryDeliveryCystic fibrosisEngineeringOligonucleotideChloride effluxHuman cellsAirway epitheliumLung tissue
2001
Gene targeting via triple-helix formation
Casey B, Glazer P. Gene targeting via triple-helix formation. Progress In Nucleic Acid Research And Molecular Biology 2001, 67: 163-192. PMID: 11525382, DOI: 10.1016/s0079-6603(01)67028-4.Peer-Reviewed Original Research