grant
Charcot-Marie-Tooth Disease Type 2E: Mechanism and therapy
Organization OHIO STATE UNIVERSITYLocation Columbus, UNITED STATESPosted 15 May 2025Deadline 30 Apr 2030
NIHUS FederalResearch GrantFY2025AAV vectorAAV-based vectorAfferent NeuronsAntimorphic mutationArray tomographyAxonAxonal TransportAxoplasmic TransportBiochemicalBiopsyCaliberCaringCell BodyCellsCellular MatrixCharcot Marie DisorderCharcot Marie Muscular AtrophyCharcot Marie Tooth DisorderCharcot Marie Tooth muscular atrophyCharcot-Marie DiseaseCharcot-Marie-ToothCharcot-Marie-Tooth DiseaseCharcot-Marie-Tooth neuropathyClinicalCultured CellsCytoplasmCytoskeletal SystemCytoskeletonDNA TherapyDNA mutationDataDegenerative Neurologic DisordersDiseaseDisorderDistalDominant NegativeDominant-Negative MutantDominant-Negative MutationDorsal Root GangliaElectron MicroscopyElectrophysiologyElectrophysiology (science)GEM modelGEMM modelGene Transfer ClinicalGenesGenetic ChangeGenetic InterventionGenetic defectGenetic mutationGenetically Engineered MouseGenomicsGoalsHMSNHereditaryHereditary Motor and Sensory NeuropathiesHeterozygoteHistologicHistologicallyHumanImageImmunofluorescenceImmunofluorescence ImmunologicImpairmentInduced pluripotent stem cell derived human neuronInduced pluripotent stem cell derived neuronsInheritedInjectionsIntermediate FilamentsInterventionInvestigatorsKI miceKineticsKnock-in MouseMedulla SpinalisMiceMice MammalsMicroscopicMissense MutationModern ManMolecularMotorMotor CellMotor NeuronsMovementMurineMusMutationNerveNerve CellsNerve ConductionNerve UnitNervous System Degenerative DiseasesNeural CellNeural ConductionNeural Degenerative DiseasesNeural degenerative DisordersNeurocyteNeurodegenerative DiseasesNeurodegenerative DisordersNeurologic Degenerative ConditionsNeuron from iPSCNeuron from induced pluripotent stem cellsNeuronal DifferentiationNeuronsNeurophysiology / ElectrophysiologyOhioPatientsPeroneal Muscular AtrophyPhysiologicPhysiologicalPolymersPost-Transcriptional Gene SilencingProtein SubunitsProteinsRNA InterferenceRNA SilencingRNAiRecombinant adeno-associated virusRecombinant adeno-associated virus (rAAV)RecombinantsReportingResearchResearch PersonnelResearchersResistanceRoleSensorySensory NeuronsSequence-Specific Posttranscriptional Gene SilencingSerotypingSpinal CordSpinal GangliaStructureSwellingTestingTherapeuticTranslationsUniversitiesViral GenesViral VectorVirusadeno-associated viral vectoradeno-associated virus vectorautosomal dominant mutationbody movementcomparable efficacycomparative efficacycompare efficacydegenerative diseases of motor and sensory neuronsdegenerative neurological diseasesdesigndesigningdisease modeldisorder modeldominant genetic mutationdominant mutationdorsal root ganglionelectrophysiologicalexperienceflexibilityflexiblegene editing methodgene editing methodologygene editing strategygene editing techniquesgene repair therapygene therapygene-based therapygene-editing approachgenetic therapygenetically engineered mouse modelgenetically engineered murine modelgenome mutationgenomic therapyheterozygosityhiPSChiPSC-derived neuronshuman iPShuman iPSChuman iPSC-derived sensory neuronhuman induced pluripotent cellhuman induced pluripotent stem cell derived sensory neuronhuman induced pluripotent stem cellshuman inducible pluripotent stem cellshuman inducible stem cellsiPSiPS neuronsiPSCiPSC derived-neuronsiPSC-derived human neuroniPSCsimagingimaging approachimaging based approachinduced human pluripotent stem cellsinduced pluripotent cellinduced pluripotent stem cellinduced pluripotent stem cell neuronsinducible pluripotent cellinducible pluripotent stem cellinducible pluripotent stem cell derived human neuroninducible pluripotent stem cell derived human sensory neuroninterestintracellular skeletonknock-downknockdownknockin micemissense single nucleotide polymorphismmissense single nucleotide variantmissense variantmotoneuronmouse modelmurine modelmutantneural cell bodyneurodegenerative illnessneurofilamentneuromuscular functionneuronalneuronal cell bodyneurons derived from induced pluripotent stem cellsneurons differentiated from human induced pluripotent stem cellsneurons differentiated from induced pluripotent stem cellsneuropathic muscular atrophyoverexpressoverexpressionpolymerpolymericpost-natal developmentpostnatal developmentpre-clinicalpreclinicalpreventpreventingpromoterpromotorrAAVrecombinant AAVreconstructionresistantscaffoldscaffoldingsocial rolesomasuccesstranslationvector
Description preview
ABSTRACT
Neurofilaments (NFs) are abundant cytoskeletal polymers in neurons that function as space-filling structures to
expand axonal caliber, which is a critical determinant of axonal conduction velocity. NFs assemble in the
neuronal soma and are transported into axons, where they accumulate excessively in many neurodegenerative
diseases,…
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