grant
Molecular and Clinical Manifestations of Matrix and Aggregate Myopathies
Organization NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKELocation UNITED STATES
NIHUS FederalResearch GrantFY20250-11 years oldASO therapeuticsASO therapyASO treatmentAddressAdolescentAdolescent YouthAffectAfferent NeuronsAnimal ModelAnimal Models and Related StudiesAntimorphic mutationAntisense Oligonucleotide TherapyAtrophicAtrophyBiological MarkersBiopsyBreathingCOL6A1COL6A2CRISPRCRISPR/Cas systemCase StudyCausalityCell Communication and SignalingCell SignalingCell modelCell-Extracellular MatrixCellular modelCentronuclear myopathyChildChild YouthChildhoodChildren (0-21)ClinicClinicalClinical TrialsClinical assessmentsClustered Regularly Interspaced Short Palindromic RepeatsCodeCoding SystemCohort StudiesCollaborationsCollagen Type VICompensationConcurrent StudiesCongenital Myasthenia GravisCongenital Myasthenic SyndromesDNA TherapyDNA mutationDedicationsDevelopmentDiseaseDisorderDominant NegativeDominant-Negative MutantDominant-Negative MutationDoseDrug KineticsECMEngineeringEtiologyEvaluationExtracellular MatrixFHL1FHL1 geneFHL1BFLH1AFour and a half LIM domains 1GaitGene TransferGene Transfer ClinicalGene therapy trialGenesGeneticGenetic ChangeGenetic InterventionGenetic defectGenetic mutationGenomic approachGenomicsGoalsGrowth AgentsGrowth FactorGrowth SubstancesHumanImageImaging DeviceImaging InstrumentImaging ToolImmunomodulationInfrastructureInternationalIntima CollagenIntracellular Communication and SignalingJointsKYO-TLIM protein SLIMMERLaboratoriesMGC111107MR ImagingMR TomographyMRIMRIsMagnetic Resonance ImagingMediatingMedical Imaging, Magnetic Resonance / Nuclear Magnetic ResonanceModalityModern ManMolecularMuscleMuscle DiseaseMuscle DisordersMuscle TissueMuscular DiseasesMutationMyopathic ConditionsMyopathic Diseases and SyndromesMyopathic disease or syndromeMyopathyMyotubular MyopathyNCATSNGS MethodNGS systemNMR ImagingNMR TomographyNational Center for Advancing Translational SciencesNational Institutes of HealthNatural HistoryNatural regenerationNeuromuscular DiseasesNeuromuscular Junction DiseasesNeuromuscular Junction DisordersNeuromuscular Transmission DisordersNuclear Magnetic Resonance ImagingOrphan DiseaseOutcome MeasureOutcome StudyPainPainfulPathway interactionsPatientsPharmaceutical AgentPharmaceuticalsPharmacokineticsPharmacologic SubstancePharmacological SubstancePhasePhenotypePiezo 2Piezo 2 ion channelPiezo2PositionPositioning AttributePost-Transcriptional Gene SilencingProprioceptionProteins Growth FactorsProtocolProtocols documentationRNA InterferenceRNA SeqRNA SilencingRNA SplicingRNA sequencingRNAiRNAseqRare DiseasesRare DisorderReceptor ProteinRegenerationResearchRespiratory AspirationRespiratory InspirationRespiratory MusclesSLIM1SensorySensory NeuronsSequence-Specific Posttranscriptional Gene SilencingSignal TransductionSignal Transduction SystemsSignalingSplicingSyndromeTechnologyTestingTherapeuticTissue ModelTouchTouch sensationTranscriptTranslatingTranslationsType VI (Intima) CollagenUnited States National Institutes of HealthValidationVentilatory MusclesVisualizationWorkX-linked centronuclear myopathyX-linked congenital recessive muscle hypotrophy with central nucleiX-linked myotubular myopathyX-linked recessive centronuclear myopathyX-linked recessive myotubular myopathyXMPMAZeugmatographyanti-sense oligonucleotide druganti-sense oligonucleotide therapyanti-sense oligonucleotide treatmentanti-sense therapyantisense drugantisense oligonucleotide therapeuticantisense therapeuticsantisense therapybA535K18.1base editingbio-markersbiologic markerbiological signal transductionbiomarkercase reportcausal allelecausal genecausal mutationcausal variantcausationcausative mutationcausative variantclinical outcome measuresclinical translationclinically translatablecohortcollagen 6collagen Type 6collagen VIcongenital muscular dystrophycongenital myopathydevelopmentaldisease causationdisease modeldisorder modelearly onsetentire genomeexomeexosuitfallsfirst in manfirst-in-humanfull genomegene repair therapygene therapeuticsgene therapygene transfer trialgene-based therapeuticgene-based therapeuticsgene-based therapygenes therapeuticgenes therapeuticsgenetic therapygenome mutationgenome sequencinggenomic datagenomic datasetgenomic effortgenomic strategygenomic therapygiant axonal neuropathyhumanized micehumanized mouseiPSiPSCiPSCsimagingimmune modulationimmune regulationimmunologic reactivity controlimmunomodulatoryimmunoregulationimmunoregulatoryimprovedinduced pluripotent cellinduced pluripotent stem cellinducible pluripotent cellinducible pluripotent stem cellinspirationjuvenilejuvenile humankidsloss of functionmeasurable outcomemembermodel of animalmouse modelmurine modelmuscularmuscular disordermyoneural disorderneurogeneticsneuromuscularneuromuscular degenerative disorderneuromuscular disordernext gen sequencingnext generationnext generation sequencingnextgen sequencingnovelorphan disorderoutcome measurementpathwaypediatricpharmaceuticalphenotypic dataprecision medicineprecision-based medicinepreservationprogramsreceptorregeneratesafety assessmentsensory prosthesistactile sensationtherapeutic genetooltranscriptome sequencingtranscriptomic sequencingtranslationtrial designvalidationswhole genomeyoungster
Description preview
The Neuromuscular and Neurogenetic Disorders of Childhood Section (NNDCS) is dedicated to elucidating the genetic and pathophysiologic basis of early-onset neuromuscular disorders, to exploring therapeutic approaches to these conditions and to bringing first clinical trials to patients affected by these disorders. To accomplish these goals, we are…
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