grant
CRISPRa-based rescue of sensorimotor deficits in the Scn2a+/- mouse model of autism spectrum disorder
Organization UNIVERSITY OF CALIFORNIA, SAN FRANCISCOLocation SAN FRANCISCO, UNITED STATESPosted 1 Apr 2024Deadline 31 Jan 2029
NIHUS FederalResearch GrantFY202612-20 years old21+ years oldASDAction PotentialsAcuteAdolescenceAdolescentAdolescent YouthAdultAdult HumanAffectAllelesAllelic LossAllelomorphsAnimalsAreaAutismAutistic DisorderAxonBehaviorBehavioralBiological MarkersBrainBrain Nervous SystemBrain regionCRISPR activationCRISPR activatorCRISPR based activationCRISPR gene activationCRISPR transcription activationCRISPR transcriptional activationCRISPR-Cas-9-mediated gene activationCRISPR-based gene activationCRISPR-dCAS9 ActivatorCRISPR-mediated transcriptional activationCRISPR/CAS9 activationCRISPR/CAS9 gene activationCRISPR/dCas9 activationCRISPR/dCas9-based transcriptional activationCRISPRaCell BodyCell FunctionCell PhysiologyCell ProcessCellsCellular FunctionCellular PhysiologyCellular ProcessCerebellumCodeCoding SystemCytoplasmic GranulesDNA TherapyDataDendritesDevelopmentDiseaseDisorderDysfunctionEEGEarly Infantile AutismElectrocorticogramElectroencephalogramElectroencephalographyEncephalonEnhancersExhibitsFrequenciesFunctional disorderGene ExpressionGene Transfer ClinicalGenesGeneticGenetic InterventionGoalsHigh-Throughput Nucleotide SequencingHigh-Throughput SequencingHumanImpairmentInfantile AutismInterventionKanner's SyndromeLaboratoriesLearningLinkLoss of HeterozygosityMapsMembraneMethodsMiceMice MammalsModelingModern ManMorbid ObesityMotorMurineMusNav1.2NeocortexNerve CellsNerve UnitNeural CellNeurocyteNeurodevelopmental DisorderNeurological Development DisorderNeuronsPhenotypePhysiologicPhysiologicalPhysiologyPhysiopathologyPopulationPositionPositioning AttributePrefrontal CortexPreparationProteinsPurkinje CellsPurkinje's CorpusclesPyramidal CellsReagentReflexReflex actionRisk-associated variantSCN2A proteinSensorySevere obesitySodium ChannelSodium Ion ChannelsSomatosensory CortexSpace PerceptionSpatial DiscriminationSpecificitySubcellular ProcessSynapsesSynapticSynaptic plasticitySystemTestingTherapeuticTherapeutic InterventionTouchTouch sensationTranscription ActivatorTranscription CoactivatorTranscription Factor CoactivatorTranscriptional Activator/CoactivatorTransmissionUpregulationVibrissaeVisualWhiskersWorkactivating CRISPR technologyadolescence (12-20)adulthoodassess effectivenessautism modelautism spectral disorderautism spectrum disorderautistic spectrum disorderbehavior phenotypebehavioral impairmentbehavioral phenotypingbio-markersbiologic markerbiomarkercerebellar Purkinje cellcritical developmental periodcritical perioddetermine effectivenessdetermine efficacydevelopmentaleffective interventioneffectiveness assessmenteffectiveness evaluationeffectiveness testingefficacy analysisefficacy assessmentefficacy determinationefficacy evaluationefficacy examinationelectrocorticographyevaluate effectivenessevaluate efficacyexamine effectivenessexamine efficacyexperimentexperimental researchexperimental studyexperimentsextreme obesityfunctional genomicsgene repair therapygene therapygene-based therapygenetic therapygenomic therapygranulegranule cellhomotypical cortexiPSiPSCiPSCsimpaired behaviorinduced pluripotent cellinduced pluripotent stem cellinducible pluripotent cellinducible pluripotent stem cellintervention therapyisocortexjuvenilejuvenile humanloss of functionloss of function mutationmembrane structuremodel of autism spectrum disordermouse modelmurine modelna(v)1.2neocorticalneopalliumneuralneurodevelopmental diseaseneuronalnew drug treatmentsnew drugsnew pharmacological therapeuticnew therapeuticsnew therapynext generation therapeuticsnovel drug treatmentsnovel drugsnovel pharmaco-therapeuticnovel pharmacological therapeuticnovel therapeuticsnovel therapyoculovestibular reflexpathophysiologyperceptual spatial orientationpostsynapticpreparationspromoterpromotorrepetitive behaviorresponserisk allelerisk generisk genotyperisk locirisk locusrisk variantsensory cortexsocial communication impairmentsomatosensorysomesthetic sensory cortexspatial orientationspatial perceptionsynapsesynapse functionsynaptic functiontactile sensationtransmission processvestibo-ocular reflexesvestibulo-occular systemvestibulo-ocular reflexvestibulo-oculomotor reflexvestibuloocular reflexesvoltage
Description preview
Title: CRISPRa-based rescue of sensorimotor deficits in the Scn2a+/- mouse model of autism
spectrum disorder
Project Summary: Heterozygous loss-of-function mutations in the sodium channel gene SCN2A
are strongly associated with autism spectrum disorder. SCN2A encodes the neuronal sodium
channel NaV1.2, which contributes to membrane excitability in…
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