grant
Shared Resource Core
Organization VIRGINIA COMMONWEALTH UNIVERSITYLocation RICHMOND, UNITED STATESPosted 25 Sept 2025Deadline 31 Aug 2030
NIHUS FederalResearch GrantFY2025AccelerationAddressAdoptedAntisense AgentAntisense OligonucleotidesAssayAwarenessBenchmarkingBest Practice AnalysisBioassayBioinformaticsBiologic ModelsBiological AssayBiological MarkersBiological ModelsBody SystemBody TissuesCataractCell LineCellLineClinicalClinical ResearchClinical StudyClinical TrialsCommunitiesComplexConduct Clinical TrialsConsultationsDNADataDeoxyribonucleic AcidDevelopmentDiseaseDisorderDystrophia MyotonicaElementsEmbryonic Muscle CellsEnsureEventFacultyFibroblastsFutureGene ExpressionGenesGenetic MedicineGenomicsGoalsImpairmentIndividualIndustryInterruptionInvestigatorsLengthLibrariesLinkMeasuresMethodsModel SystemMolecularMorbidityMorbidity - disease rateMultiple PartnersMuscleMuscle RelaxationMuscle TissueMuscle WeaknessMuscle functionMuscle relaxation phaseMuscular WeaknessMyoblastsMyotoniaMyotonia AtrophicaMyotonia DystrophicaMyotonic DystrophyNatureNon-Polyadenylated RNAOrgan SystemPathogenicityPatientsPharmaceutical AgentPharmaceuticalsPharmacologic SubstancePharmacological SubstancePhasePhenotypePopulationPrecursor Muscle CellsProcessProtocolProtocols documentationRNARNA Gene ProductsRNA SplicingRNA-Binding ProteinsReporterResearchResearch PersonnelResearch ResourcesResearchersResource SharingResourcesRibonucleic AcidRunningSamplingServicesShort interfering RNASmall Interfering RNASplicingSteinert DiseaseStrains Cell LinesTestingTherapeuticTherapeutic TrialsTissuesTrainingTranscriptUniversitiesantisense oligobenchmarkbio-markersbiobankbiologic markerbiomarkerbiomarker developmentbiorepositorycataractogenesiscataractous lensesclinical developmentclinical phenotypeconsultationcultured cell linedesigndesigningdetermine efficacydevelopmentaldisease modeldisorder modeldystrophic myotoniaearly clinical trialearly onsetearly phase clinical trialefficacy analysisefficacy assessmentefficacy determinationefficacy evaluationefficacy examinationevaluate efficacyexamine efficacyexperiencegenetic analysisgenomic datagenomic datasetiPSiPSCiPSCsimprovedindexinginduced pluripotent cellinduced pluripotent stem cellinducible pluripotent cellinducible pluripotent stem cellindustrial partnershipindustry partnerindustry partnershipinterestinternet portalinternet resourcemortalitymouse modelmurine modelmuscle strengthmuscularnew drug treatmentsnew drugsnew pharmacological therapeuticnew therapeuticsnew therapynext generation therapeuticsnovel drug treatmentsnovel drugsnovel pharmaco-therapeuticnovel pharmacological therapeuticnovel therapeuticsnovel therapyon-line compendiumon-line portalon-line resourceonline compendiumonline portalonline resourceopen sourcepharmaceuticalphenotypic datapre-clinicalpre-clinical developmentpreclinicalpreclinical developmentprogramssiRNAsmall moleculesuccesstherapeutic agent developmenttherapeutic developmenttranscriptomicstreatment strategyweb portalweb resourceweb siteweb-based portalweb-based resourcewebsitewiki
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Modified Project Summary/Abstract Section
The myotonic dystrophies share a common pathogenic mechanism and have similar but non overlapping phenotypes. The discovery of the pathogenic mechanism, along with advances in genetic medicine, have rapidly accelerated the development of potential therapies for these conditions. The investigators are…
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