grant
Expression of dystrophins with enhanced function
Organization UNIVERSITY OF WASHINGTONLocation SEATTLE, UNITED STATESPosted 22 Jan 2025Deadline 31 Dec 2029
NIHUS FederalResearch GrantFY2026AAV vectorAAV-based vectorASO therapeuticsASO therapyASO treatmentAddressAffectAgingAnimal ModelAnimal Models and Related StudiesAntisense AgentAntisense Oligonucleotide TherapyAntisense OligonucleotidesAttentionBypassCRISPRCRISPR/Cas systemCachecticCachexiaCapsidCell Communication and SignalingCell SignalingClinicClinicalClinical TrialsCloningClustered Regularly Interspaced Short Palindromic RepeatsComplementary DNACoupledDNA Double Strand BreakDNA Sequence RearrangementDNA TherapyDNA editorDNA mutationDataDoseDose LimitingDucheneDuchenneDuchenne muscular dystrophyDuchenne-Griesinger syndromeDysfunctionDystrophinDystrophin-Related ProteinDystrophin-Related Protein 1Ellis-van Creveld (EvC) syndromeEnsureEnzyme GeneEnzymesExerciseFDA approvedFaceFunctional disorderGene AlterationGene DeliveryGene MutationGene Transfer ClinicalGeneralized GrowthGenesGenetic ChangeGenetic DiseasesGenetic InterventionGenetic defectGenetic mutationGoalsGrowthHumanHybridsImmune responseInjuryInsertional MutagenesisIntervening Protein SequenceIntracellular Communication and SignalingLengthLength of LifeLinkLongevityMediatingMethodsModern ManMuscleMuscle DiseaseMuscle DisordersMuscle TissueMuscle satellite cellMuscular DiseasesMuscular DystrophiesMutationMyodystrophicaMyodystrophyMyopathic ConditionsMyopathic Diseases and SyndromesMyopathic disease or syndromeMyopathyNeuromuscular DiseasesPatientsPhysiopathologyPoint MutationPrevalenceProductionProgenitor CellsProtein IntronsProteinsPseudohypertrophic Muscular DystrophySafetySerious Adverse EventSerotypingSevere Adverse EventSignal TransductionSignal Transduction SystemsSignalingStriated MusclesStructureSyndromeSystemTechnologyTestingTherapeuticTimeTissue GrowthToxic effectToxicitiesTraumaTreatment EfficacyTreatment outcomeUTRN ProteinUtrophinVariantVariationViralViral VectorX-linked dilated cardiomyopathyX-linked muscular dystrophyX-linked recessive muscular dystrophyadeno-associated viral vectoradeno-associated virus vectoranimal dataanti-sense oligonucleotide druganti-sense oligonucleotide therapyanti-sense oligonucleotide treatmentanti-sense therapyantisense drugantisense oligoantisense oligonucleotide therapeuticantisense therapeuticsantisense therapybase editingbenign X-linked recessive muscular dystrophybiological signal transductioncDNAchildhood pseudohypertrophic muscular dystrophyclassic X-linked recessive muscular dystrophyclinical translationclinically translatablecomparable efficacycomparativecomparative efficacycompare efficacydesigndesigningexon skippingfacesfacialfunctional improvementgene defectgene editing methodgene editing methodologygene editing strategygene editing techniquesgene editorgene repair therapygene replacementgene therapygene-based therapygene-editing approachgenetic conditiongenetic disordergenetic recessivegenetic therapygenome editorgenome mutationgenomic rearrangementgenomic therapygenotyped patientshost responseimmune system responseimmunoresponseimprove functionimprovedimproved functional outcomesin vivoinjuriesinteinintervention efficacymicro-dystrophinmicrodystrophinmild X-linked recessive muscular dystrophymodel of animalmuscle dystrophymuscle progenitormuscle progenitor cellmuscle stem cellmuscularmuscular disordermutantmutant allelemyoneural disorderneuromuscular degenerative disorderneuromuscular disordernew technologynovelnovel technologiesontogenypathophysiologypostmitoticpreventpreventingprogressive muscular dystrophy of childhoodpseudohypertrophic adult muscular dystrophypseudohypertrophic muscular paralysisrecessive genetic traitrecessive traitreconstitutereconstitutionrepairrepairedresponsesarcopeniasarcopenicsatellite cellscRNA sequencingscRNA-seqserious adverse experienceserious adverse reactionsingle cell RNA-seqsingle cell RNAseqsingle cell expression profilingsingle cell transcriptomic profilingsingle-cell RNA sequencingspatial and temporalspatial temporalspatiotemporalstem cellstherapeutic efficacytherapy adverse effecttherapy efficacytooltreatment adverse effectvector
Description preview
Project Summary/Abstract
Duchenne muscular dystrophy (DMD) is an X-linked, lethal recessive genetic disorder resulting from mutations
in the DMD gene, which encodes the protein dystrophin (Dys). The 2.2 MB gene displays the highest new
mutation rate of any human gene, reflecting the high DMD prevalence (1:5,000). This large size coupled with…
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