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Investigating the role of the Unfolded Protein Response in Genetic Dilated Cardiomyopathy
Organization STANFORD UNIVERSITYLocation STANFORD, UNITED STATESPosted 30 Sept 2024Deadline 29 Jan 2026 ⚠️
NIHUS FederalResearch GrantFY2025ATF-4AssayBAG3BAG3 geneBCL2-Associated Athanogene 3Basal Transcription FactorBasal transcription factor genesBioassayBiological AssayCRISPR approachCRISPR based approachCRISPR methodCRISPR methodologyCRISPR techniqueCRISPR technologyCRISPR toolsCRISPR-CAS-9CRISPR-based methodCRISPR-based techniqueCRISPR-based technologyCRISPR-based toolCRISPR/CAS approachCRISPR/Cas methodCRISPR/Cas technologyCRISPR/Cas9CRISPR/Cas9 technologyCalcium Ion SignalingCalcium SignalingCardiacCardiac DiseasesCardiac DisordersCardiac Muscle CellsCardiac MyocytesCardiocyteCas nuclease technologyCell BodyCell Communication and SignalingCell LineCell SignalingCellLineCellsCellular MatrixCellular StressCellular Stress ResponseChIP assayClustered Regularly Interspaced Short Palindromic Repeats approachClustered Regularly Interspaced Short Palindromic Repeats methodClustered Regularly Interspaced Short Palindromic Repeats methodologyClustered Regularly Interspaced Short Palindromic Repeats techniqueClustered Regularly Interspaced Short Palindromic Repeats technologyCongestive CardiomyopathyCytoskeletal SystemCytoskeletonDNA TherapyDNA mutationDataDefectDesmosomesDevelopmentDilated CardiomyopathyDiseaseDisorderDysfunctionER stressExhibitsFailureFunctional disorderGene AlterationGene MutationGene TranscriptionGene Transfer ClinicalGeneral Transcription Factor GeneGeneral Transcription FactorsGenesGeneticGenetic ChangeGenetic InterventionGenetic ModelsGenetic TranscriptionGenetic defectGenetic mutationGenotypeGoalsHeartHeart DiseasesHeart Muscle CellsHeart failureHeart myocyteHereditaryHumanImpairmentIn VitroInheritedIntracellular Communication and SignalingIonsLeftLinkMacula AdherensMediatingMediatorMitochondriaModern ManMolecularMutationMyocardial depressionMyocardial dysfunctionNode of BizzozeroOntologyPathogenesisPathologic ProcessesPathological ProcessesPathologyPathway interactionsPatientsPhenocopyPhenotypePhysiopathologyPlayPost-Transcriptional Gene SilencingPrevalenceProteinsPumpRNA ExpressionRNA InterferenceRNA SilencingRNAiReactionResearchRoleSequence-Specific Posttranscriptional Gene SilencingSignal PathwaySignal TransductionSignal Transduction SystemsSignalingSourceSpot DesmosomeStrains Cell LinesTestingTherapeuticTranscriptionTranscription Factor Proto-OncogeneTranscription factor genesTranslatingUpregulationVariantVariationVentricularWorkaberrant folded proteinaberrant folded proteinsaberrant protein foldingabnormal folded proteinabnormal folded proteinsabnormal protein foldingabnormal protein homeostasisabnormal proteostasisactivating transcription factor 4biological adaptation to stressbiological signal transductioncardiac dysfunctioncardiac failurecardiac functioncardiac myocytes differentiated from induced pluripotent stem cellcardiomyocytecell stresschromatin immunoprecipitationcohortcultured cell linedefective proteostasisdevelopmentaldisease modeldisorder modelendoplasmic reticulum stressexperimentexperimental researchexperimental studyexperimentsfunction of the heartgene defectgene repair therapygene therapygene-based therapygenetic therapygenome editinggenome mutationgenomic editinggenomic therapyheart disorderheart dysfunctionheart functioniPSiPS cell derived cardiomyocytesiPSCiPSC derived cardiomyocytesiPSCsimprovedin vivoin vivo Modelinduced pluripotent cellinduced pluripotent stem cellinduced pluripotent stem cell derived cardiac myocytesinduced pluripotent stem cell derived cardiomyocytesinduced pluripotent stem cells derived from patientsinduced pluripotent stem cells from patientsinducible pluripotent cellinducible pluripotent stem cellinducible pluripotent stem cell derived cardiac myocytesinducible pluripotent stem cells derived cardiomyocytesinsoluble aggregateintracellular skeletonknock-downknockdownmisfolded proteinmisfolded proteinsmitochondrialmouse modelmurine modelmutant allelenew drug targetnew druggable targetnew pharmacotherapy targetnew therapeutic targetnew therapy targetnovelnovel drug targetnovel druggable targetnovel pharmacotherapy targetnovel therapeutic targetnovel therapy targetoverexpressoverexpressionpathologic protein foldingpathophysiologypathwaypatient derived human iPSpatient derived human iPSCpatient derived human induced pluripotent stem cellpatient derived iPSpatient derived iPSCpatient derived induced pluripotent cellspatient derived induced pluripotent stem cellspatient-derived pluripotent stem cellspharmacologicphospholambanprotein aggregateprotein aggregationprotein homeostasisprotein homeostasis declineprotein homeostasis deficiencyprotein homeostasis dysfunctionprotein homeostasis failureprotein homeostasis lossprotein misfoldingproteostasisproteostasis declineproteostasis defectproteostasis deficiencyproteostasis dysfunctionproteostasis dysregulationproteostasis failureproteostasis impairmentproteostasis lossproteotoxic proteinproteotoxinreaction; crisisresponsesmall moleculesocial rolestress responsestress; reactionsudden cardiac deaththerapeutic targettooltranscription factor
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Description preview
Project Summary
Dilated Cardiomyopathy (DCM) is the leading cause of heart failure. Characterized by left ventricular dilation
and impaired contractility, DCM results in pump failure and arrhythmogenesis leading directly to heart failure
and sudden cardiac death. There are more than 50 genes implicated in DCM development that each produce a
common…
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