2024
Prenatal Therapy for Congenital Diaphragmatic Hernia and Myelomeningocele: Advances in Particle-Based Delivery
Rivero R, Stitelman D. Prenatal Therapy for Congenital Diaphragmatic Hernia and Myelomeningocele: Advances in Particle-Based Delivery. Current Stem Cell Reports 2024, 10: 51-57. DOI: 10.1007/s40778-024-00237-8.Peer-Reviewed Original ResearchCongenital diaphragmatic herniaDiaphragmatic herniaAnimal modelsIntra-amniotic injectionLong-term functional outcomeParticle-based deliveryFetal therapyPrenatal therapySpinal defectsTarget specific tissuesCo-morbiditiesCongenital diseaseFunctional outcomesTreatment deliveryStructural diseaseClinical translationMyelomeningoceleTherapyHerniaSpecific tissuesTissueDiseaseDeliveryPrenatallyParticle delivery
2022
Targeted 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
In vivo self-assembled small RNAs as a new generation of RNAi therapeutics
Fu Z, Zhang X, Zhou X, Ur-Rehman U, Yu M, Liang H, Guo H, Guo X, Kong Y, Su Y, Ye Y, Hu X, Cheng W, Wu J, Wang Y, Gu Y, Lu S, Wu D, Zen K, Li J, Yan C, Zhang C, Chen X. In vivo self-assembled small RNAs as a new generation of RNAi therapeutics. Cell Research 2021, 31: 631-648. PMID: 33782530, PMCID: PMC8169669, DOI: 10.1038/s41422-021-00491-z.Peer-Reviewed Original ResearchConceptsRNAi therapeuticsRNAi therapyVivo siRNA deliveryEGFR/KRASSiRNA deliveryVivo deliverySynthetic siRNAsLung cancerHost liverPotent target geneSecretory exosomesTherapeutic valueCircuit moduleArtificial vehiclesGenetic circuitsTherapyMultiple tissuesSiRNAsCritical targetPrinciple strategyTissueSpecific tissuesTherapeuticsDeliveryExosomes
2018
Regulatory T cells in autoimmune disease
Dominguez-Villar M, Hafler DA. Regulatory T cells in autoimmune disease. Nature Immunology 2018, 19: 665-673. PMID: 29925983, PMCID: PMC7882196, DOI: 10.1038/s41590-018-0120-4.Peer-Reviewed Original ResearchConceptsAutoimmune diseasesTreg cellsRegulatory T cell biologyRegulatory T cellsNon-immune cellsTreg cell plasticityTreg cell biologyNew therapeutic strategiesT cell biologyTreg cell instabilityDisease outcomeT cellsTherapeutic strategiesDiseaseCell plasticityCell biologyCellsAutoimmunityPathogenesisSpecific tissues
2016
Novel Biologic Therapy for Untreated Diseases of Vascular Calcification
Braddock D, Kavanagh D, Li X. Novel Biologic Therapy for Untreated Diseases of Vascular Calcification. Blood 2016, 128: 2326. DOI: 10.1182/blood.v128.22.2326.2326.Peer-Reviewed Original ResearchConnective tissue diseasePlasma PPiTissue calcificationPseudoxanthoma elasticumABCC6 mutationsTissue diseaseVascular calcificationMembrane transport proteinsKnockout miceStem cell progenitorsMineralization inhibitorPotent mineralization inhibitorNovel biologic therapiesElastic fiber alterationsTransport proteinsFusion proteinVivo experimental evidenceCell progenitorsFunction mutationsBiologic therapyUntreated diseaseArterial calcificationNeonatal diseaseSpecific tissuesCommon disorderThe Steroidogenic Acute Regulatory Protein (StAR) Is Regulated by the H19/let-7 Axis
Men Y, Fan Y, Shen Y, Lu L, Kallen AN. The Steroidogenic Acute Regulatory Protein (StAR) Is Regulated by the H19/let-7 Axis. Endocrinology 2016, 158: 402-409. PMID: 27813675, PMCID: PMC5413078, DOI: 10.1210/en.2016-1340.Peer-Reviewed Original ResearchConceptsSteroidogenic acute regulatory proteinRegulatory proteinsAcute regulatory proteinPost-transcriptional levelRate-limiting stepMicroRNA let-7H19/letRegulation of reproductionMurine cell linesLet-7Overexpression of H19Long noncoding RNA H19Regulation of steroidogenesisCellular growthSpecific tissuesNovel mechanismMultiple rolesNovel targetRNA H19StAR expressionExpression variesCell linesRegulationH19ProteinExpression of ribosomopathy genes during Xenopus tropicalis embryogenesis
Robson A, Owens ND, Baserga SJ, Khokha MK, Griffin JN. Expression of ribosomopathy genes during Xenopus tropicalis embryogenesis. BMC Developmental Biology 2016, 16: 38. PMID: 27784267, PMCID: PMC5081970, DOI: 10.1186/s12861-016-0138-5.Peer-Reviewed Original ResearchConceptsRibosome biogenesis factorsBiogenesis factorsVentral blood islandsHuman congenital diseasesCranial neural crestRNA-seq dataAntisense mRNA probesBlood islandsEmbryonic developmentDistinct tissuesNeural crestProtein productionDifferential expressionTranscript numbersMachinery componentsSpecific tissuesSpecific phenotypesRibosomopathiesSitu hybridizationMRNA probesRibosomesExpressionTissue localizationPhenotypeFactor expression
2015
Mosquito Defense Strategies against Viral Infection
Cheng G, Liu Y, Wang P, Xiao X. Mosquito Defense Strategies against Viral Infection. Trends In Parasitology 2015, 32: 177-186. PMID: 26626596, PMCID: PMC4767563, DOI: 10.1016/j.pt.2015.09.009.Peer-Reviewed Original ResearchConceptsViral infectionMosquito antiviral immunityEfficient antiviral strategiesPathological sequelaePersistent infectionAntiviral immunityArbovirus infectionViral replicationAntiviral strategiesInfectionMosquito tissuesViral diseasesGlobal healthViral propagationMosquitoesTissueNatural vectorSequelaeSpecific tissuesDiseaseImmunity
2009
A Robust Approach to Identifying Tissue-Specific Gene Expression Regulatory Variants Using Personalized Human Induced Pluripotent Stem Cells
Lee JH, Park IH, Gao Y, Li JB, Li Z, Daley GQ, Zhang K, Church GM. A Robust Approach to Identifying Tissue-Specific Gene Expression Regulatory Variants Using Personalized Human Induced Pluripotent Stem Cells. PLOS Genetics 2009, 5: e1000718. PMID: 19911041, PMCID: PMC2766639, DOI: 10.1371/journal.pgen.1000718.Peer-Reviewed Original ResearchMeSH KeywordsAllelesCell DifferentiationCell LineCells, CulturedCluster AnalysisComputational BiologyDNA, ComplementaryFlow CytometryGene Expression RegulationHuman Genome ProjectHumansInduced Pluripotent Stem CellsNucleic Acid Amplification TechniquesOrgan SpecificityRegulatory Elements, TranscriptionalReproducibility of ResultsConceptsCis-regulatory variantsPluripotent stem cellsRegulatory polymorphismsGene expressionAllele-specific gene expressionIPS cellsPrimary fibroblastsCell typesAllele-specific expressionHigh-throughput sequencingStem cellsHuman iPS cellsSkin primary fibroblastsRegulatory variantsTissue-specific variantsX chromosomeDifferentiated derivativesExpression locusCell differentiationSpecific tissuesLymphoblastoid cellsDigital-RNAPadlock probesExpressionCells
2006
Estrogen Receptor-Mediated Rapid Signaling
Moriarty K, Kim KH, Bender JR. Estrogen Receptor-Mediated Rapid Signaling. Endocrinology 2006, 147: 5557-5563. PMID: 16946015, DOI: 10.1210/en.2006-0729.Peer-Reviewed Original ResearchConceptsMembrane estrogen receptorsPlasma membrane-associated formSplice variantsMembrane-associated formTranscriptional activationMultimolecular complexesRapid signalingSignaling cascadesMolecular componentsSpecific tissuesER splice variantsMolecular levelSpecific fashionEnzymatic pathwaysEstrogen receptorEstrogen responseNuclear responsePathwayPhysiological outcomesReceptorsComplexesSignalingVariantsVascular endotheliumRapid response
2005
Immunosurveillance by γδ T Cells: Focus on the Murine System
Girardi M, Hayday AC. Immunosurveillance by γδ T Cells: Focus on the Murine System. Chemical Immunology And Allergy 2005, 86: 136-150. PMID: 15976492, DOI: 10.1159/000086658.Peer-Reviewed Original ResearchConceptsT cell subsetsCell subsetsT cellsDistinct T cell subsetsSpecific T cell subsetsT cell responsesΓδ T cellsGammadelta T cellsPhysiologic immune responseLymphoid cell typesImmune surveillanceImmune responseMouse modelCell responsesClinical settingMurine systemCell typesDifferent genetic backgroundsGenetic backgroundAssociationCell associationCellsInflammationImmunosurveillanceSpecific tissues
1983
SALT: Skin-Associated Lymphoid Tissues
Streilein J, Tigelaar R. SALT: Skin-Associated Lymphoid Tissues. 1983, 95-130. DOI: 10.1007/978-1-4613-3670-9_6.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsSkin-associated lymphoid tissueImmune responseExogenous antigensLymphoid tissueImmune systemUnique immune responseSpecific immune responseMucous membrane surfacesImmunologic apparatusLymph nodesEffector cellsImmunocompetent lymphocytesRespiratory tractEffector mechanismsImmunologic influenceOral routeNervous systemOrgan systemsAntigenPathogenic potentialSpecific tissuesTissueUnrelated tissuesGutCells
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