grant
Research Project I: AAV-CRISPR to Treat Huntington's Disease
Organization UNIVERSITY OF CALIFORNIA BERKELEYLocation BERKELEY, UNITED STATESPosted 15 May 2023Deadline 30 Apr 2028
NIHUS FederalResearch GrantFY2025AAV vectorAAV-based vectorAbscissionAcuteAdeno-Associated VirusesAllelesAllelomorphsAnimal ModelAnimal Models and Related StudiesAnimalsAssociated VirusesAutopsyBasal GangliaBasal NucleiBindingBiodistributionBody TissuesBrainBrain Nervous SystemBrain regionCRISPRCRISPR approachCRISPR based approachCRISPR methodCRISPR methodologyCRISPR techniqueCRISPR technologyCRISPR toolsCRISPR-CAS-9CRISPR-based methodCRISPR-based techniqueCRISPR-based technologyCRISPR-based toolCRISPR/CAS approachCRISPR/Cas methodCRISPR/Cas systemCRISPR/Cas technologyCRISPR/Cas9CRISPR/Cas9 technologyCapsidCas nuclease technologyClinicClinicalClinical EvaluationClinical TestingClinical TrialsClustered Regularly Interspaced Short Palindromic RepeatsClustered 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 technologyCommon Rat StrainsComplexContracting OpportunitiesContractsConvectionCorpus StriatumCorpus striatum structureCrab-Eating MacaqueCrab-Eating MonkeyCynomolgus MonkeyCynomolgus macaqueDNADNA Double Strand BreakDNA TherapyDNA mutationDataDegenerative Neurologic DisordersDeoxyribonucleic AcidDependoparvovirusDependovirusDiseaseDisorderDoseDyskinesia SyndromesEncephalonEngineeringEnsureEnzyme GeneEnzymesEvaluationExcisionExonsExtirpationFibroblastsFood and Drug AdministrationGene AlterationGene MutationGene Transfer ClinicalGenesGenetic ChangeGenetic InterventionGenetic defectGenetic mutationGenomicsGuide RNAGuidelinesHD GeneHD proteinHaplotypesHereditary DiseaseHumanHuntingtinHuntingtin ProteinHuntington ChoreaHuntington DiseaseHuntington geneHuntington proteinHuntington'sHuntington's DiseaseHuntington's disease gene productHuntingtons DiseaseIND FilingIND applicationIND packageIND submissionIT15 geneIn VitroInborn Genetic DiseasesInduced pluripotent stem cell derived neuronsInherited disorderInjectionsInvestigational DrugsInvestigational New Drug ApplicationInvestigational New DrugsLeadLengthM fascicularisM. fascicularisMR ImagingMR TomographyMRIMRIsMacaca cynomolgusMacaca fascicularisMacaca irusMagnetic Resonance ImagingMediatingMedical Imaging, Magnetic Resonance / Nuclear Magnetic ResonanceMiceMice MammalsModern ManMolecularMolecular InteractionMovement Disorder SyndromesMovement DisordersMurineMusMutationNMR ImagingNMR TomographyNervous System Degenerative DiseasesNervous System DiseasesNervous System DisorderNeural Degenerative DiseasesNeural degenerative DisordersNeurodegenerative DiseasesNeurodegenerative DisordersNeurologic Degenerative ConditionsNeurologic DisordersNeurological DisordersNeuron from iPSCNeuron from induced pluripotent stem cellsNon-Polyadenylated RNANuclear Magnetic Resonance ImagingOnset of illnessOrganPathogenicityPatientsPb elementPeripheralPersonsPoly QPre IND FDA meetingPre-IND mtgProcessProteinsR-Series Research ProjectsR01 MechanismR01 ProgramRNARNA Gene ProductsRatRats MammalsRattusRecombinantsRemovalReportingResearch GrantsResearch Project GrantsResearch ProjectsRibonucleic AcidRodentRodentiaRodents MammalsSafetySatellite VirusesSerotypingSeveritiesSiteSpecific qualifier valueSpecifiedStriate BodyStriatumSurgical RemovalSystemTestingThalamic structureThalamusTherapeuticTissuesToxic effectToxicitiesToxicologyTranslatingUSFDAUnited States Food and Drug AdministrationZeugmatographyadeno associated virus groupadeno-associated viral vectoradeno-associated virus vectorbehavior testbehavioral testbrain tissuecell transformationclinical testdegenerative diseases of motor and sensory neuronsdegenerative neurological diseasesdelivery vectordelivery vehicledesigndesigningdetermine efficacydisease onsetdisease phenotypedisorder onseteffective therapyeffective treatmentefficacy analysisefficacy assessmentefficacy determinationefficacy evaluationefficacy examinationefficacy testingevaluate efficacyexamine efficacyexperimentexperimental researchexperimental studyexperimentsfeasibility testingfirst in manfirst-in-humangRNAgain of function mutationgene defectgene repair therapygene therapygene-based therapygenetic therapygenome editinggenome mutationgenomic editinggenomic therapyheavy metal Pbheavy metal leadhereditary disorderheritable disorderiPS neuronsiPSC derived-neuronsinborn errorinduced pluripotent stem cell neuronsinherited diseasesinherited genetic diseaseinherited genetic disorderinteresting transcript 15knock-downknockdownlong-tailed macaquemeetingmeetingsmodel of animalmouse modelmurine modelmutantmutant allelenecropsynerve cell deathnerve cell lossneurodegenerative illnessneurogeneticsneurological diseaseneuron cell deathneuron cell lossneuron deathneuron lossneuronal cell deathneuronal cell lossneuronal deathneuronal lossneurons derived from induced pluripotent stem cellsneurons differentiated from induced pluripotent stem cellsnon-human primatenonhuman primatenovelpolyQpolyglutaminepostmortempre-IND consultationpre-IND discussionpre-IND meetingpre-Investigational New Drug meetingpre-clinicalpreclinicalprogramsprogressive neurodegenerationputamenrepairrepairedresearch clinical testingresectionscreeningscreeningsstriatalthalamictissue/cell culturetransduction efficiencytransformed cellsvector
Description preview
PROJECT SUMMARY / ABSTRACT
Background: Huntington's disease (HD) is a fatal, dominant neurogenetic disorder caused by
polyglutamine repeat expansion in exon 1 of the HD gene encoding huntingtin (HTT). It is
estimated that 1−3 in every 10,000 persons (nearly 30,000 in the USA) have HD. Patients suffer
from progressive neurodegeneration in the basal…
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