grant
In vivo investigation of PKD1 upstream open reading frames as a therapeutic target in autosomal dominant polycystic kidney disease
NIHUS FederalResearch GrantFY20265' Untranslated Regions5'UTR80K-H proteinADPKDAbdominal PainAdult Polycystic Kidney DiseaseAffectAllelesAllelomorphsAntisense AgentAntisense OligonucleotidesAssayAutosomal Dominant Polycystic KidneyAutosomal Dominant Polycystic Kidney DiseaseAwardBAC cloneBACsBacterial Artificial ChromosomesBasal Transcription FactorBasal transcription factor genesBioassayBiologicalBiological AssayCRISPRCRISPR/Cas systemCell BodyCell surfaceCellsChaperoneClustered Regularly Interspaced Short Palindromic RepeatsCodonCodon NucleotidesCystCystic kidneyDNA AlterationDNA Sequence AlterationDNA mutationDataDevelopmentDiseaseDisorderDominant Polycystic Kidney DiseaseDoseESKDESRDEnd stage renal failureEnd-Stage Kidney DiseaseEnd-Stage Renal DiseaseEndoplasmic ReticulumEpidermal CystEpidermal Inclusion CystEpidermoid CystEpithelial cystErgastoplasmFDA approvedFGF-stimulated p90G19P1 proteinGeneral Transcription Factor GeneGeneral Transcription FactorsGeneralized GrowthGenesGenetic AlterationGenetic ChangeGenetic defectGenetic mutationGenomicsGrowthHepatectomyHepatic CystHepatic TransplantationHorn CystHumanImmunoblottingImpairmentIn VitroInclusion CystInitiation CodonInitiator CodonInterventionInvestigationKeratin CystKeratinizing CystKeratinous CystKidneyKidney CystKidney FailureKidney InsufficiencyKidney Urinary SystemLaboratoriesLiquid substanceLiverLiver CystLiver GraftingLiver TransplantLuciferase ImmunologicLuciferasesMessenger RNAMiceMice MammalsMicroRNAsModelingModern ManMolecular ChaperonesMouse Cell LineMurineMusMutationNucleotidesORFsOpen Reading FramesPKD1 genePKD1 proteinPKD2 proteinPRKCSHPRKCSH proteinPathogenicityPatientsPeptidesPersonsPilar CystPopulationProductionProtein BiosynthesisProtein Coding RegionProteinsPublishingRegulationRenal CystRenal FailureRenal InsufficiencyRepressionRibosomal Peptide BiosynthesisRibosomal Protein BiosynthesisRibosomal Protein SynthesisRoleSebaceous CystSequence AlterationSeverity of illnessSiteSomatic MutationStart CodonTestingTherapeuticTissue GrowthTranscription Factor Proto-OncogeneTranscription factor genesTranslatingTranslationsVasopressin ReceptorWestern BlottingWestern ImmunoblottingWorkXBP1XBP1 geneantagonismantagonistantisense oligobiologicclinical diagnosiscongenital cystic liver diseasecystic liver diseasedevelopmentaldisease severitydosageeffective therapyeffective treatmentfibrocystic liver diseasefluidgenome mutationgenome scalegenome-widegenomewidegenomic alterationhepatic body systemhepatic organ systemhumanized micehumanized mousein vivoliquidliver transplantationmRNAmRNA ExpressionmRNA Leader SequencesmiRNAmouse modelmurine modelnew approachesnovelnovel approachesnovel strategiesnovel strategyoligonucleotide deliveryoligonucleotide drugoligonucleotide therapeuticsoligonucleotide therapiesoligonucleotide treatmentontogenyoverexpressoverexpressionpatient subclasspatient subclusterpatient subgroupspatient subpopulationspatient subsetspatient subtypespcy proteinpolycystic breakpoint proteinpolycystic kidney disease 1 (autosomal dominant)polycystic kidney disease 1 proteinpolycystic kidney disease 2 proteinpolycystic liver diseasepolycystin 1polycystin 2preventpreventingprotein blottingprotein expressionprotein kinase substrate 80K-Hprotein kinase substrate 80KD protein, heavy chainprotein synthesisrenalrepairrepairedsocial rolesomatic varianttherapeutic targettolvaptantranscription factortranslationtranslational opportunitiestranslational potential
Description preview
Project Summary:
Autosomal dominant polycystic kidney disease (ADPKD) affects over 12 million people worldwide resulting in
fluid-filled cysts in the kidney and liver and 5-10% of all kidney failure. The one FDA-approved therapy provides
only a modest delay in ongoing growth of cysts in the kidney and liver that can progress to kidney failure and…
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