grant
CMT4A and CMT2K Gene Replacement Therapy with AAV9/GDAP1 in Rats
Organization UT SOUTHWESTERN MEDICAL CENTERLocation DALLAS, UNITED STATESPosted 15 Feb 2026Deadline 31 Jan 2028
NIHUS FederalResearch GrantFY2026Adeno-Associated VirusesAffectAge MonthsAllelesAllelomorphsAnimal ModelAnimal Models and Related StudiesAntimorphic mutationAutopsyBehavioralCLN1CLN1 geneCLN1 proteinCLN5CLN5 geneCLN5 proteinCell BodyCellsCerebroatrophic HyperammonemiaCerebrospinal FluidCharcot Marie DisorderCharcot Marie Muscular AtrophyCharcot Marie Tooth DisorderCharcot Marie Tooth muscular atrophyCharcot-Marie DiseaseCharcot-Marie-ToothCharcot-Marie-Tooth DiseaseCharcot-Marie-Tooth neuropathyClinicalClinical TrialsCollaborationsCommon Rat StrainsDNA TherapyDataDependoparvovirusDependovirusDevelopmentDiffuse Globoid Body SclerosisDiseaseDisorderDominant NegativeDominant-Negative MutantDominant-Negative MutationDoseElectrophysiologyElectrophysiology (science)EquilibriumEuthanasiaFeedbackFutureGDAP proteinGalactosylceramidase Deficiency DiseaseGene AlterationGene MutationGene Transfer ClinicalGenesGenetic InterventionGloboid LeukodystrophyGloboid cell leukodystrophyGoalsHand functionsHeterozygoteHistologicHistologicallyHumanIND FilingIND applicationIND packageIND submissionInjectionsInterventionIntrathecal InjectionsInvestigational DrugsInvestigational New Drug ApplicationInvestigational New DrugsKO miceKnock-out MiceKnockout MiceKrabbe DiseaseKrabbe leukodystrophyLaboratoriesLegLifeLongitudinal StudiesLongitudinal SurveysMediatingMedulla SpinalisMercy KillingMiceMice MammalsModelingModern ManMonitorMurineMusMuscle AtrophyMuscle WeaknessMuscular AtrophyMuscular WeaknessNatureNerve CellsNerve ConductionNerve UnitNeural CellNeural ConductionNeurilemma CellNeurilemmal CellNeurocyteNeuromuscular DiseasesNeuronsNeuropathyNeurophysiology / ElectrophysiologyNull MousePathogenicityPatientsPeripheral NervesPeripheral Nervous SystemPeroneal Muscular AtrophyPersonsPhenotypeRandomizedRatRats MammalsRattusRett DisorderRett SyndromeSafetySchwann CellsSensorySpinal CordTestingTherapeuticTherapy Clinical TrialsToxic effectToxicitiesTranslatingTranslationsUsing handsVariantVariationWalkingWorkadeno associated virus grouparmautosomal dominant mutationautosomebalancebalance functionbehavior phenotypebehavioral phenotypingbeta galactocerebrosidase deficiencycerebral spinal fluidclinical translationclinically translatabledesigndesigningdetermine efficacydevelopmentaldiffuse globoid cell cerebral sclerosisdisease phenotypeearly onsetefficacy analysisefficacy assessmentefficacy determinationefficacy evaluationefficacy examinationelectrophysiologicalevaluate efficacyexamine efficacyexperiencefirst in manfirst-in-humangain of function mutationgalactocerebrosidase (GALC) deficiencygalactocerebrosidase deficiencygalactosylceramide beta-galactosidase deficiencygalactosylceramide deficiencygalactosylceramide lipidosisgalactosylsphingosine lipidosisganglioside-induced differentiation-associated proteingene defectgene repair therapygene replacement therapygene therapygene-based therapygenetic therapygenomic therapygiant axonal neuropathygloboid cell cerebral sclerosisgloboid cell sclerosisheterozygositylong-term studylongitudinal outcome studieslongitudinal research studyloss of function mutationmodel of animalmouse modelmurine modelmuscle breakdownmuscle degradationmuscle deteriorationmuscle lossmuscle wastingmutant allelemyoneural disordernecropsyneuromuscular degenerative disorderneuromuscular disorderneuronalneuronal 5 ceroid-lipofuscinosisneuropathicpostmortempre-clinicalpreclinicalprotein functionpsychosine lipidosisrandomisationrandomizationrandomly assignedspinal fluidsuccesstransgene expressiontranslationtreatment groupvector
Description preview
PROJECT SUMMARY/ABSTRACT
Charcot–Marie–Tooth (CMT) neuropathies are a genetically and phenotypically heterogeneous group of
disorders caused by pathogenic variants in over 100 different genes. Ganglioside-induced differentiation-
associated protein 1 (GDAP1) gene mutations cause various forms of CMT including CMT4A and CMT2K.
CMT4A is a severe,…
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