grant
Project 1: Therapeutic Gene Editing for Spinal Muscular Atrophy (Trailblazer)
Organization JACKSON LABORATORYLocation BAR HARBOR, UNITED STATESPosted 16 May 2023Deadline 30 Apr 2028
NIHUS FederalResearch GrantFY2025AccelerationAgeAllelesAllelomorphsAnimalsAran-Duchenne diseaseBiodistributionBody TissuesCRISPRCRISPR/Cas systemCell BodyCellsCessation of lifeClinicClinical TrialsClustered Regularly Interspaced Short Palindromic RepeatsCodeCoding SystemCombined Modality TherapyCommunicationCompensationCruveilhier diseaseDNA TherapyDataDeathDoseDrug TherapyEthnic GroupEthnic PeopleEthnic PopulationEthnic individualEthnicity PeopleEthnicity PopulationExonsFunctional impairmentGene ConversionGene Transfer ClinicalGenesGeneticGenetic InterventionGenomeGenomicsGoalsIND FilingIND applicationIND packageIND submissionIn VitroIndustry StandardInfant MortalityInfant Mortality TotalInvestigational DrugsInvestigational New Drug ApplicationInvestigational New DrugsInvestigatorsLeadLengthMessenger RNAMiceMice MammalsModelingModificationMonitorMotor CellMotor Neuron DiseaseMotor NeuronsMultimodal TherapyMultimodal TreatmentMurineMusNervous System DiseasesNervous System DisorderNeurologic DisordersNeurological DisordersNucleotidesOutcomePalsyParalysedPatientsPb elementPharmacological StudyPharmacological TreatmentPharmacology StudyPharmacotherapyPhenotypePlegiaPositionPositioning AttributeProteinsProtocolProtocols documentationPublishingR-Series Research ProjectsR01 MechanismR01 ProgramRNA SplicingRegulationReproducibilityResearch GrantsResearch PersonnelResearch Project GrantsResearch ProjectsResearch ResourcesResearchersResourcesRouteSMN expressionSMN geneSMN gene product (SMA)SMN proteinSMN protein (spinal muscular atrophy)SMN protein expressionSMN1SMN1 geneSMN2SMN2 geneSafetySamplingSchemeSpecificitySpinalSpinal Muscular AtrophySplicingTestingTherapeuticTherapeutic Gene EditingTissuesToxic effectToxicitiesToxicologyTranslatingTreatment ProtocolsTreatment RegimenTreatment ScheduleWorkadverse consequenceadverse outcomeagesbase editingclinical significanceclinically significantcombination therapycombined modality treatmentcombined treatmentdeath among infantsdeath in first year of lifedeath in infancydeath in infantsdegenerative disorder of motor neuronsdetermine efficacydrug interventiondrug treatmentefficacy analysisefficacy assessmentefficacy determinationefficacy evaluationefficacy examinationefficacy studyethnic subgroupethnicity groupevaluate efficacyexamine efficacyexon skippinggene repair therapygene therapygene-based therapygene-editing therapygenetic therapygenome editinggenome editing based therapygenome editing therapygenome editing treatmentgenome editing-based therapeuticsgenomic editinggenomic therapyheavy metal Pbheavy metal leadin vivoinfant deathinfant demiseinfantile deathlead candidatelife spanlifespanmRNAmanufacturemortality in infantsmotoneuronmouse modelmulti-modal therapymulti-modal treatmentmurine modelneonateneurological diseaseoverexpressoverexpressionparalysisparalyticpharmaceutical interventionpharmacological interventionpharmacological therapypharmacology interventionpharmacology treatmentpharmacotherapeuticspre-clinicalpre-clinical developmentpreclinicalpreclinical developmentprogramsrestorationsurvival motor neuron genesurvival motor neuron gene productsurvival motor neuron proteinsurvival of motor neuron 1survival of motor neuron 2therapeutic editingtherapeutic genome editingtherapy optimizationtooltreatment optimization
Description preview
PROJECT SUMMARY PROJECT 1 (SMA)
Spinal Muscular Atrophy (SMA) is a progressive motor neuron disease (MND) and the leading genetic cause of
infant mortality across all ethnic groups. SMA is caused by the homozygous loss of the essential survival motor
neuron 1 (SMN1) gene and while one or more copies of the highly similar SMN2 gene partially…
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