grant
Alternative Splicing and Development of Small Molecule Therapeutics in CAG Expansion Spinocerebellar Ataxias
Organization STATE UNIVERSITY OF NEW YORK AT ALBANYLocation ALBANY, UNITED STATESPosted 1 Dec 2023Deadline 30 Nov 2028
NIHUS FederalResearch GrantFY2025AccelerationAddressAlternate SplicingAlternative RNA SplicingAlternative SplicingAnimalsAtaxiaAtaxyBioavailabilityBiological AvailabilityBiological MarkersCell LineCellLineClinical EvaluationClinical TestingClinical TrialsCoordination ImpairmentDNA mutationDataDegenerative DisorderDevelopmentDifferential Gene ExpressionDiseaseDisease OutcomeDisorderDominantly-Inherited Spinocerebellar AtaxiasDyssynergiaDystrophia MyotonicaEquilibriumEventExpression SignatureFDA approvedFibroblastsFutureGene Expression ProfileGene TranscriptionGenesGeneticGenetic ChangeGenetic TranscriptionGenetic defectGenetic mutationGenomicsHereditaryHumanImmunoblottingIn vivo analysisInduced pluripotent stem cell derived neuronsInheritedInvestigationLeadLinkLongitudinal StudiesMJD1 proteinMeasuresMiceMice MammalsMicrosatellite MarkersMicrosatellite RepeatsMicrosatellitesModern ManMolecularMovementMurineMusMutationMyotonia AtrophicaMyotonia DystrophicaMyotonic DystrophyNatural ProductsNerve CellsNerve UnitNervous System DiseasesNervous System DisorderNeural CellNeurocyteNeurologic DisordersNeurological DisordersNeuron from iPSCNeuron from induced pluripotent stem cellsNeuronal DysfunctionNeuronsNon-Polyadenylated RNAOnset of illnessPathogenesisPathologicPathologyPathway interactionsPatientsPb elementPhenotypePhysiologic AvailabilityPoly QPopulationProductionProteinsPublishingRNARNA ExpressionRNA Gene ProductsRNA SeqRNA SplicingRNA sequencingRNAseqRT-PCRReporterReverse Transcriptase Polymerase Chain ReactionRibonucleic AcidSCA3SafetySpinocerebellar AtaxiasSpinocerebellar AtrophiesSplicingSteinert DiseaseStrains Cell LinesSystemTestingTherapeuticTimeTissue-Specific Differential Gene ExpressionTissue-Specific Gene ExpressionTranscriptTranscriptionTreatment EfficacyWestern BlottingWestern ImmunoblottingWorkataxin-3balancebalance functionbehavior phenotypebehavioral phenotypingbio-markersbiologic markerbiomarkerbody movementchemical libraryclinic readyclinical readyclinical testcultured cell linedegenerative conditiondegenerative diseasedevelop therapydevelopmentaldifferential expressiondifferentially expresseddisease onsetdisorder onsetdystrophic myotoniaexperiencegene expression patterngene expression signaturegenome mutationglobal gene expressionglobal transcription profileheavy metal Pbheavy metal leadiPS neuronsiPSC derived-neuronsimprovedin vivo evaluationin vivo testinginduced pluripotent stem cell neuronsinsightintervention developmentintervention efficacylead candidatelong-term studylongitudinal outcome studiesmouse modelmurine modelnaturally occurring productneural dysfunctionneurological diseaseneuronalneurons derived from induced pluripotent stem cellsneurons differentiated from induced pluripotent stem cellsnew drug targetnew druggable targetnew markernew pharmacotherapy targetnew therapeutic targetnew therapy targetnovelnovel biomarkernovel drug targetnovel druggable targetnovel markernovel pharmacotherapy targetnovel therapeutic targetnovel therapy targetpathwaypolyQpolyglutaminepre-clinical efficacypre-clinical studypre-clinical therapypreclinical efficacypreclinical studypreclinical therapyprotein blottingratiometricresearch clinical testingreverse transcriptase PCRscreeningscreeningssmall moleculesmall molecule librariessmall molecule therapeuticstherapeutic agent developmenttherapeutic biomarkertherapeutic developmenttherapeutic efficacytherapeutic evaluationtherapeutic markertherapeutic testingtherapy developmenttherapy efficacytranscriptional differencestranscriptional profiletranscriptional signaturetranscriptometranscriptome sequencingtranscriptomic sequencingtranscriptomicstreatment development
Description preview
Abstract
The spinocerebellar ataxias (SCAs) are a genetically heterogeneous group of rare, dominantly inherited
neurological disorders characterized by progressive ataxia. Although the genetic causes of SCAs are diverse,
multiple SCAs (1,2,3,6,7 & 12) are caused by CAG expansion mutations and they share the production of CAG
expansion RNAs and in…
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