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 optionTargeted Suppression of miRNA-33 Using pHLIP Improves Atherosclerosis Regression
Zhang X, Rotllan N, Canfrán-Duque A, Sun J, Toczek J, Moshnikova A, Malik S, Price NL, Araldi E, Zhong W, Sadeghi MM, Andreev OA, Bahal R, Reshetnyak YK, Suárez Y, Fernández-Hernando C. Targeted Suppression of miRNA-33 Using pHLIP Improves Atherosclerosis Regression. Circulation Research 2022, 131: 77-90. PMID: 35534923, PMCID: PMC9640270, DOI: 10.1161/circresaha.121.320296.Peer-Reviewed Original ResearchConceptsMiR-33Gene expressionNature of miRNAsSingle-cell RNA sequencing analysisSingle-cell RNA transcriptomicsAnti-miRNA technologiesRNA sequencing analysisExpression of miRNAsRNA transcriptomicsNew therapeutic opportunitiesEntire pathwayMiRNA therapeuticsAtherosclerotic plaque macrophagesHuman diseasesMiRNAsSequencing analysisSpecific tissuesMetabolic tissuesTargeted suppressionMiR-33 inhibitionProtective miRNAsNumerous diseasesPharmacological inhibitionLipid accumulationTarget effects
2021
Novel RNA Interference (RNAi)-Based Nanomedicines for Treating Viral Infections
Maes N, Zeller S, Kumar P. Novel RNA Interference (RNAi)-Based Nanomedicines for Treating Viral Infections. 2021, 223-264. DOI: 10.1201/9781003125259-8.Peer-Reviewed Original ResearchJapanese encephalitis virusNon-target tissuesHuman papillomavirusPediatric encephalitisTherapeutic modalitiesMajor safety concernNovel RNA interferenceViral infectionReplication-competent lentivirusClinical testingEncephalitis virusInfectious diseasesMajor causeSafety concernsDiseaseVector mobilizationRibonucleic acid interferenceTarget effectsRNA interferenceTargets of microRNAsEncephalitisPapillomavirusVaccineInfectionFlaviviruses
2020
Cytoskeletal Drugs Modulate Off-Target Protein Folding Landscapes Inside Cells
Davis CM, Gruebele M. Cytoskeletal Drugs Modulate Off-Target Protein Folding Landscapes Inside Cells. Biochemistry 2020, 59: 2650-2659. PMID: 32567840, DOI: 10.1021/acs.biochem.0c00299.Peer-Reviewed Original ResearchConceptsCytoskeletal drugsPhosphoglycerate kinaseActin filamentsDynamic cytoskeletal networksEffects of cytoskeletonProtein energy landscapesOff-target proteinsOpposite responseCytoskeletal networkProtein stabilityCellular milieuProtein-like sequencesVariable major protein-like sequenceOverall cell volumeCytoskeletonCell migrationEnergy landscapeMacromolecular crowdingMacromolecular crowdersProteinNonspecific surface interactionsTarget effectsMicrotubulesCytoplasmCells
2016
Antibody–Drug Conjugates (ADCs) in Clinical Development
McLaughlin J, LoRusso P. Antibody–Drug Conjugates (ADCs) in Clinical Development. 2016, 321-344. DOI: 10.1002/9781119060727.ch13.Peer-Reviewed Original ResearchAntibody-drug conjugatesCytotoxic agentsMonoclonal antibodiesLocal immune responseFavorable safety profileConventional cytotoxic chemotherapyConventional cytotoxic agentsDifferent antibody–drug conjugatesImmune-stimulating agentsAnti-neoplastic agentsCytotoxic chemotherapySafety profileCancer patientsIL-2Clinical trialsImmune responseClinical developmentOncologist's abilityImproved efficacyPhase IDrug conjugatesAntibodiesEfficacyTarget effectsToxicity
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
2014
Bioreactor technologies to support liver function in vitro
Ebrahimkhani MR, Neiman JA, Raredon MS, Hughes DJ, Griffith LG. Bioreactor technologies to support liver function in vitro. Advanced Drug Delivery Reviews 2014, 69: 132-157. PMID: 24607703, PMCID: PMC4144187, DOI: 10.1016/j.addr.2014.02.011.Peer-Reviewed Original ResearchConceptsSmall molecule drugsNucleic acid therapyNew experimental modelPrediction of metabolismImmunologic homeostasisAcid therapyLiver functionDrug discoveryMolecule drugsLiver physiologyExperimental modelLong-term liver-specific functionDisease modelsDrug developmentMolecular therapeuticsTarget effectsTherapeuticsWide spectrumIndirect targetsPathophysiologyTherapyLiver-specific functions
2013
Dynamin triple knockout cells reveal off target effects of commonly used dynamin inhibitors
Park RJ, Shen H, Liu L, Liu X, Ferguson SM, De Camilli P. Dynamin triple knockout cells reveal off target effects of commonly used dynamin inhibitors. Journal Of Cell Science 2013, 126: 5305-5312. PMID: 24046449, PMCID: PMC3828596, DOI: 10.1242/jcs.138578.Peer-Reviewed Original ResearchConceptsDynamin 1Dynamin inhibitorTKO cellsPeripheral membrane rufflingDouble knockoutDynamin 3 geneClathrin-mediated endocytosisTriple-knockout cellsDynamin-dependent processFluid-phase endocytosisDyngo-4aMembrane fissionMembrane rufflingDKO cellsDynamin 2Knockout cellsLow-level expressionCell physiologyDynaminEndocytosisTarget effectsDKO fibroblastsGenesCellsKO fibroblasts
2012
YM155 sensitivity in pediatric acute lymphoblastic leukemia.
Chang B, Jemal A, Tyner J, Thayer M, Muschen M, Druker B. YM155 sensitivity in pediatric acute lymphoblastic leukemia. Journal Of Clinical Oncology 2012, 30: 9555-9555. DOI: 10.1200/jco.2012.30.15_suppl.9555.Peer-Reviewed Original ResearchPediatric acute lymphoblastic leukemiaAcute lymphoblastic leukemiaLymphoblastic leukemiaPhase I/II studyNCI standard riskStandard intensive chemotherapyProtein expressionSubset of patientsAdditional therapeutic agentsB-cell malignanciesYM155 treatmentIntensive chemotherapyII studyPhospho-flow cytometryPrimary patientsCombination therapyAnnexin V bindingTarget effectsNovel agentsPossible efficacyHigh riskStandard riskIsobologram analysisMultiple protein expressionMalignant cells
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