grant
Mechanisms of Neurodegeneration in KIF5A ALS/FTD
Organization UNIVERSITY OF PITTSBURGH AT PITTSBURGHLocation PITTSBURGH, UNITED STATESPosted 1 Aug 2023Deadline 31 Jul 2028
NIHUS FederalResearch GrantFY2025ALS patientsAdaptor ProteinAdaptor Protein GeneAdaptor Signaling ProteinAdaptor Signaling Protein GeneAffectAgeAmyotrophic Lateral SclerosisAmyotrophic Lateral Sclerosis Motor Neuron DiseaseAmyotrophic Lateral Sclerosis patientsAmyotrophic lateral sclerosis and frontotemporal degenerationAmyotrophic lateral sclerosis and frontotemporal dementiaApplications GrantsAutoregulationAxonAxonal TransportAxoplasmic TransportBehaviorBehavioralBindingBiochemicalBiologyBody TissuesBrainBrain Nervous SystemC-terminalCRISPR 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 technologyCas nuclease technologyCell BodyCellsCellular MatrixCessation of lifeClustered 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 technologyComplexCytoplasmic GranulesCytoskeletal SystemCytoskeletonDNA mutationDataDeathDefectDegenerative Neurologic DisordersDevelopmentDevelopment PlansDiseaseDisorderDistalDoctor of MedicineDoctor of PhilosophyDysfunctionEncephalonExclusionExonsFTD dementiaFTD/ALSFTLD/ALSFluorescence Light MicroscopyFluorescence MicroscopyFoundationsFrontal Temporal DementiaFrontotemporal DementiaFrontotemporal Lobar Degeneration/Amyotrophic lateral sclerosisFunctional disorderFundingFutureGEM modelGEMM modelGehrig's DiseaseGeneticGenetic ChangeGenetic EngineeringGenetic Engineering BiotechnologyGenetic Engineering Molecular BiologyGenetic defectGenetic mutationGenetically Engineered MouseGliosisGoalsGrant ProposalsHealthHomeostasisHyperactivityImageImaging ProceduresImaging TechnicsImaging TechniquesImmunoblottingIn VitroInduced pluripotent stem cell derived human neuronKinesinKnowledgeLabelLanguageLength of LifeLongevityLou Gehrig DiseaseLysosomesM.D.MaintenanceManuscriptsMedulla SpinalisMentorsMentorshipMiceMice MammalsMicro-tubuleMicrotubulesMitochondriaMolecular InteractionMorphologyMotilityMotorMotor CellMotor Neuron DiseaseMotor NeuronsMurineMusMutationMyoneural JunctionNerve CellsNerve DegenerationNerve UnitNervous System Degenerative DiseasesNeural CellNeural Degenerative DiseasesNeural degenerative DisordersNeuritesNeurocyteNeurodegenerative DiseasesNeurodegenerative DisordersNeurologic Degenerative ConditionsNeuromuscular JunctionNeuron DegenerationNeuronsNon-Polyadenylated RNAOrganellesPathogenesisPathogenicityPathologyPatientsPatternPh.D.PhDPhysiciansPhysiological HomeostasisPhysiopathologyPost-Transcriptional Gene SilencingPost-Translational Modification Protein/Amino Acid BiochemistryPost-Translational ModificationsPost-Translational Protein ModificationPost-Translational Protein ProcessingPosttranslational ModificationsPosttranslational Protein ProcessingPreparationProductionProtein ModificationProteinsProteomicsPublicationsRNARNA Gene ProductsRNA InterferenceRNA SilencingRNA SplicingRNAiRecombinant DNA TechnologyRecordsRegulationRegulatory ProteinResearchResearch ResourcesResourcesRibonucleic AcidRoleScientific PublicationScientistSequence-Specific Posttranscriptional Gene SilencingSiteSlideSolidSpinal CordSplice-Junction MutationSplice-Site MutationSplicingStructureSynapsesSynapticTAR DNA-binding protein 43TDP-43TDP43TechniquesTestingTherapeuticTimeTissuesTrainingTransmission Electron MicroscopyUniversitiesWestern BlottingWestern ImmunoblottingWorkadapter proteinage associatedage correlatedage dependentage linkedage relatedage specificagedagesamyotrophic lateral sclerosis with frontotemporal dementiaamyotrophic lateral sclerosis/FTLDamyotrophic lateral sclerosis/frontotemporal dementiaamyotrophic lateral sclerosis/ftdcareer developmentcausal allelecausal genecausal mutationcausal variantcausative mutationcausative variantcognitive functioncohortconfocal imagingdegenerative diseases of motor and sensory neuronsdegenerative disorder of motor neuronsdegenerative neurological diseasesdevelopmentalfront temporal dementiafrontal lobe dementiafrontotemporal dementia-amyotrophic lateral sclerosisfrontotemporal lobar degeneration dementiafrontotemporal lobar dementiafrontotemporal lobar dementia amyotrophic lateral sclerosisfrontotemporal lobe degeneration associated with dementiagain of functiongain of function mutationgenetic regulatory proteingenetically engineeredgenetically engineered mouse modelgenetically engineered murine modelgenome mutationgranulehiPSChiPSC-derived neuronshuman diseasehuman 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 cellsiPSiPSCiPSC-derived human neuroniPSCsimagingin vivoinduced human pluripotent stem cellsinduced pluripotent cellinduced pluripotent stem cellinducible pluripotent cellinducible pluripotent stem cellinducible pluripotent stem cell derived human neuroninducible pluripotent stem cell derived human sensory neuroninsightinsoluble aggregateintracellular skeletonlive cell imagelive cell imaginglive cellular imagelive cellular imagingmitochondrialmotoneuronmouse modelmurine modelmutantnerve cell deathnerve cell lossneural degenerationneurodegenerationneurodegenerativeneurodegenerative illnessneurofilamentneurological degenerationneuron cell deathneuron cell lossneuron deathneuron lossneuronalneuronal cell deathneuronal cell lossneuronal deathneuronal degenerationneuronal lossneurons differentiated from human induced pluripotent stem cellsnovelpathophysiologypreparationsprogramsprotein TDP-43protein TDP43protein aggregateprotein aggregationprotein blottingrational designregulatory gene productskillssmall moleculesocial rolesuper high resolutionsuperresolutionsynapsetooltraffickingultra high resolution
Description preview
Amyotrophic Lateral Sclerosis (ALS) is a fatal disease characterized by the dysfunction and death of motor
neurons. Some ALS patients develop widespread neuronal damage leading to Frontotemporal Dementia (FTD),
which is characterized by progressive behavioral change and language decline. Significant overlap exists
between ALS and FTD regarding the…
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