grant
Development of in Vivo Base Editing as a Genetic Treatment for Spinal Muscular Atrophy
Organization UNIVERSITY OF PITTSBURGH AT PITTSBURGHLocation PITTSBURGH, UNITED STATESPosted 1 Feb 2024Deadline 31 Jan 2029
NIHUS FederalResearch GrantFY2026AddressAdeno-Associated VirusesAlternate SplicingAlternative RNA SplicingAlternative SplicingAntisense AgentAntisense OligonucleotidesAran-Duchenne diseaseBody TissuesCRISPR 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 technologyClustered 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 technologyCollaborationsCommunitiesCruveilhier diseaseDNADNA AlterationDNA Sequence AlterationDNA mutationDeoxyribonucleic AcidDependoparvovirusDependovirusDevelopmentDiseaseDisorderDoseExonsFamilyFutureGenesGeneticGenetic AlterationGenetic ChangeGenetic defectGenetic mutationGoalsHealthHumanInfant MortalityInfant Mortality TotalInjectionsInternationalK01 AwardK01 MechanismK01 ProgramKnowledgeLaboratoriesLaboratory ResearchLengthLifeMentored Research Scientist Development AwardMentored Training AwardMentorsMiceMice MammalsModern ManMurineMusMutationNeuromuscular DiseasesNeuromuscular researchNon-Polyadenylated RNAPathologicPathway interactionsPatientsPenetrancePhenotypePhysiologyPositionPositioning AttributePreclinical dataProductionRNARNA Gene ProductsResearchResearch Scientist Development AwardRibonucleic AcidRouteSMN gene product (SMA)SMN proteinSMN protein (spinal muscular atrophy)SMN1SMN1 geneSMN2SMN2 geneSequence AlterationSeveritiesSpinal Muscular AtrophySystemTechnologyTherapeuticTissuesTraining ActivityTranscriptTranslational ResearchTranslational ScienceWorkWritingadeno associated virus groupantisense oligobase editingbase editorcareercareer developmentconferenceconventiondeath among infantsdeath in first year of lifedeath in infancydeath in infantsdetermine efficacydevelopmentalefficacy analysisefficacy assessmentefficacy determinationefficacy evaluationefficacy examinationevaluate efficacyexamine efficacyexon skippingexperimentexperimental researchexperimental studyexperimentsgene replacementgenome editinggenome mutationgenomic alterationgenomic editingglobal gene expressionglobal transcription profileimprovedin vivoinfant deathinfant demiseinfantile deathmeetingmeetingsmortality in infantsmouse modelmurine modelmyoneural disorderneurogeneticsneuromuscularneuromuscular degenerative disorderneuromuscular disordernew approachesnew drug treatmentsnew drugsnew pharmacological therapeuticnew therapeuticsnew therapynext generation therapeuticsnovelnovel approachesnovel drug treatmentsnovel drugsnovel pharmaco-therapeuticnovel pharmacological therapeuticnovel strategiesnovel strategynovel therapeuticsnovel therapyparalogparalogous genepathwaypre-clinicalpreclinicalpreclinical findingspreclinical informationprogramsprotein expressionskillssmall moleculesuccesssummitsurvival motor neuron gene productsurvival motor neuron proteinsurvival of motor neuron 1survival of motor neuron 2symposiasymposiumtraining moduletranscriptometranslation researchtranslational investigation
Description preview
Abstract
Spinal muscular atrophy (SMA) is a devastating neuromuscular disease and a leading genetic cause of infantile
death worldwide. Despite exciting progress in the neuromuscular field that has resulted in novel therapies, there
remains no permanent cure for SMA. Therefore, developing a permanent treatment that treats the underlying
genetic…
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