grant
Gene Therapy Delivery for Age-related Neurodegenerative Diseases
Organization UT SOUTHWESTERN MEDICAL CENTERLocation DALLAS, UNITED STATESPosted 1 Jun 2023Deadline 29 Feb 2028
NIHUS FederalResearch GrantFY202521+ years oldAAV deliveredAAV deliveryAAV vectorAAV-based deliveryAAV-based vectorAAV-based viral deliveryAAV-mediated deliveryAAV9 deliveryAAV9 mediated deliveryAAV9 vector deliveryAAV9 viral deliveryAAV9 virus to deliverAD dementiaAdeno-Associated VirusesAdeno-associated-virus-based deliveryAdultAdult HumanAffectAgeAlzheimer Type DementiaAlzheimer disease dementiaAlzheimer sclerosisAlzheimer syndromeAlzheimer'sAlzheimer's DiseaseAlzheimers DementiaAnimal ModelAnimal Models and Related StudiesAnimalsBBB crossingBBB disruptionBiochemicalBloodBlood - brain barrier anatomyBlood Reticuloendothelial SystemBlood-Brain BarrierBrainBrain Nervous SystemBrain regionCapsidCausalityCell BodyCellsCephalicCerebrospinal FluidClinical TreatmentClinical TrialsCoupledCranialDNA TherapyDataDegenerative Neurologic DisordersDependoparvovirusDependovirusDiseaseDisorderDistalDorsal Root GangliaDoseEncephalonEngineeringEtiologyExposure toFocused UltrasoundGene DeliveryGene TransferGene Transfer ClinicalGenetic InterventionGenetic MaterialsGoalsGrantHemato-Encephalic BarrierHepatotoxic effectHepatotoxicityHind BrainHistopathologyHumanImmune responseImmunizeInjectionsInsertional MutagenesisInterphase CellInterventionIntrathecal InjectionsIntravenousKineticsLiver ToxicityMT-bound tauMediatingMedulla SpinalisMethodsMiceMice MammalsMicroRNAsModern ManMurineMusNerve CellsNerve UnitNervous System Degenerative DiseasesNervous System DiseasesNervous System DisorderNeural CellNeural Degenerative DiseasesNeural degenerative DisordersNeurocyteNeurodegenerative DiseasesNeurodegenerative DisordersNeurologic Degenerative ConditionsNeurologic DisordersNeurological DisordersNeuronsNon-dividing CellNondividing CellOutcomePathogenesisPathologicPrimary Senile Degenerative DementiaProceduresRecombinant adeno-associated virusRecombinant adeno-associated virus (rAAV)ResearchResting CellRhombencephalonRiskRouteSafetySerotypingSiteSkullSpecificitySpinal CordSpinal GangliaTauopathiesTestingTherapeuticToxic effectToxic effect on liver cellsToxicitiesTranslatingTraumaTropismViralViral VectorWeightWorkabnormal tauadeno associated virus groupadeno-associated viral vectoradeno-associated viral vector 9 deliveryadeno-associated viral vector deliveryadeno-associated virus 9 deliveryadeno-associated virus deliveryadeno-associated virus mediated deliveryadeno-associated virus vectoradenovirus mediated deliveryadulthoodage associated neurodegenerationage associated neurodegenerative diseaseage associated neurodegenerative disorderage dependent neurodegenerationage dependent neurodegenerative conditionage dependent neurodegenerative diseaseage dependent neurodegenerative disorderage related neurodegenerationage-driven neurodegenerative disordersage-related neurodegenerative diseaseage-related neurodegenerative disorderagesaging associated neurodegenerationaging associated neurodegenerative diseaseaging related neurodegenerationaging related neurodegenerative diseaseaging related neurodegenerative disorderbehavior testbehavioral testblood-brain barrier crossingblood-brain barrier disruptionbloodbrain barrierbloodbrain barrier crossingbloodbrain barrier disruptionbrain parenchymacausationcerebral spinal fluidclinical interventionclinical therapycostcraniumdegenerative diseases of motor and sensory neuronsdegenerative neurological diseasesdelivered with AAVdelivery vectordelivery vehicledelivery with AAVdisease causationdorsal root gangliongene repair therapygene therapeuticsgene therapygene-based therapeuticgene-based therapeuticsgene-based therapygene-based treatmentgene-directed therapygene-targeted therapygene-targeted treatmentgenes therapeuticgenes therapeuticsgenetic therapygenomic therapyhepatic toxicityhepatoxicityhindbrainhost responseimmune system responseimmunoresponseimprovedknock-downknockdownmanufacturemiRNAmicrotubule associated protein tau mutationmicrotubule bound taumicrotubule-associated protein tau mutationmicrotubule-bound taumodel of animalmouse modelmurine modelmutant taumutation in microtubule associated protein taumutation in microtubule-associated protein tauneurodegenerative illnessneurological diseaseneuronalneuropathologic tauneuropathological tauneutralizing antibodynew approachesnovelnovel approachesnovel strategiesnovel strategyparticlepathogenic taupathogenic tau gene mutationpathological change in tauprimary degenerative dementiapromoterpromotorrAAVrecombinant AAVsenile dementia of the Alzheimer typespinal fluidtautau Proteinstau abnormalitytau associated neurodegenerationtau associated neurodegenerative processtau driven neurodegenerationtau factortau induced degenerationtau induced neurodegenerationtau intronic mutationtau mediated neurodegenerationtau mutationtau neurodegenerative diseasetau neuropathologytau pathological changetau pathologytau pathophysiologytau proteinopathytau related neurodegenerationtau-induced pathologytauopathic neurodegenerative disordertauopathytherapeutic genetherapeutic outcometherapeutically effectivetherapy outcometransduction efficiencytransgene expressiontrial regimentrial treatmentvectorweightsτ Proteinsτ mutation
Description preview
Project Summary/Abstract
Age-related neurodegenerative disorders, such as Alzheimer’s disease and related tauopathies, are a major
therapeutic challenge. There are critical brain regions implicated in disease pathogenesis and overtime the
whole or a large portion of the brain is affected. Therefore, both a focal and broad brain treatment approach…
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