grant
Pilot of a new non-viral vector for therapeutic nucleic acid delivery to the CNS
Organization MASSACHUSETTS INSTITUTE OF TECHNOLOGYLocation CAMBRIDGE, UNITED STATESPosted 1 Feb 2026Deadline 31 Jan 2028
NIHUS FederalResearch GrantFY202621+ years old7S Gamma GlobulinAD and related dementiaAD dementiaAD related dementiaAD therapyAD treatmentADRDAddressAdultAdult HumanAffectAlzheimer Type DementiaAlzheimer disease dementiaAlzheimer disease treatmentAlzheimer sclerosisAlzheimer syndromeAlzheimer treatmentAlzheimer'sAlzheimer's DiseaseAlzheimer's Disease and its related dementiasAlzheimer's and related dementiasAlzheimer's dementia and related dementiaAlzheimer's dementia or related dementiaAlzheimer's disease and related dementiaAlzheimer's disease and related disordersAlzheimer's disease and related forms of dementiaAlzheimer's disease or a related dementiaAlzheimer's disease or a related disorderAlzheimer's disease or related dementiaAlzheimer's disease related dementiaAlzheimer's disease therapyAlzheimer's therapyAlzheimers DementiaAmericanAmmon HornAnimal ModelAnimal Models and Related StudiesAntibodiesAntibody TherapyAntisense AgentAntisense OligonucleotidesArtificial nano particlesArtificial nanoparticlesBBB crossingBar CodesBathingBathsBiodistributionBiologicalBlood - brain barrier anatomyBlood-Brain BarrierBody TissuesBrainBrain Nervous SystemBrain regionBypassCNS DiseasesCNS disorderCare GiversCaregiversCell BodyCellsCentral Nervous System DiseasesCentral Nervous System DisordersCerebellomedullary CisternChargeClinicalClinical TreatmentCommon Rat StrainsConfocal MicroscopyCornu AmmonisDegenerative Neurologic DisordersDevelopmentDiseaseDisorderDoctor of PhilosophyDrug DeliveryDrug Delivery SystemsDrug KineticsEncephalonEndosomesEngineeringExhibitsFlow CytofluorometriesFlow CytofluorometryFlow CytometryFlow MicrofluorimetryFlow MicrofluorometryFluorescence Activated Cell Sorting FractionationFluorescence-Activated Cell SortingFluorescence-Activated Cell SortingsFormulationGene ExpressionGenesGeometryGoalsHealthHemato-Encephalic BarrierHippocampusHortega cellIgGImaging ProceduresImaging TechnicsImaging TechniquesImmunoglobulin GIn VitroLeadLibrariesLigandsLipid ChemistryLipidsMessenger RNAMicrogliaModalityMolecularMolecular WeightMorbidityNervous System Degenerative DiseasesNervous System DiseasesNervous System DisorderNeural Degenerative DiseasesNeural degenerative DisordersNeurodegenerative DiseasesNeurodegenerative DisordersNeurologic Degenerative ConditionsNeurologic DisordersNeurological DisordersNon-Viral VectorNucleic AcidsOligoOligonucleotidesPathway interactionsPatientsPb elementPerformancePh.D.PhDPharmacokineticsPrimary Senile Degenerative DementiaPropertyQOLQuality of lifeRatRats MammalsRattusReceptosomesReproducibilityResearchRodentRodentiaRodents MammalsRouteSamplingShort interfering RNASmall Interfering RNASpecificityStaining methodStainsStructureSurface PropertiesSystemTechniquesTechnologyTherapeuticTissuesValidationadulthoodantibody based therapiesantibody treatmentantibody-based therapeuticsantibody-based treatmentantisense oligobarcodebiocompatibilitybiologicbiomaterial compatibilityblood-brain barrier crossingbloodbrain barrierbloodbrain barrier crossingbrain cellburden of diseaseburden of illnesscell typecisterna magnaclinical interventionclinical relevanceclinical therapyclinical translationclinically relevantclinically translatablecurative interventioncurative therapeuticcurative therapycurative treatmentsdegenerative diseases of motor and sensory neuronsdegenerative neurological diseasesdelivery vectordelivery vehicledensitydesigndesigningdevelopmentaldisease burdeneffective therapyeffective treatmentengineered nano particleengineered nanoparticlefabrication costflow cytophotometryfluorescence imagingfluorescent imaginggitter cellheavy metal Pbheavy metal leadhiPSChigh rewardhigh riskhippocampalhuman iPShuman iPSChuman induced pluripotent cellhuman induced pluripotent stem cellshuman inducible pluripotent stem cellshuman inducible stem cellsimmunotoxicityin vitro activityin vivoinduced human pluripotent stem cellsinhibitorlipophilicitymRNAmanufacturemanufacturing costmanufacturing processmembermesogliamicroglial cellmicrogliocytemodel of animalmouse modelmurine modelnano meter scalenano meter sizednano particlenano-sized particlenanometer scalenanometer sizednanoparticlenanoscalenanosized particleneurodegenerative illnessneurological diseasenew drug treatmentsnew drugsnew pharmacological therapeuticnew therapeuticsnew therapynext generation therapeuticsnonviral vectornovel drug treatmentsnovel drugsnovel pharmaco-therapeuticnovel pharmacological therapeuticnovel therapeuticsnovel therapynucleic acid deliverynucleic acid therapynucleic acid-based therapeuticsoligospathwaypatient populationperivascular glial cellpre-clinicalpreclinicalprimary degenerative dementiarational designsenile dementia of the Alzheimer typesiRNAsmall moleculesmall molecule therapeuticssuccesstherapeutic nucleic acidstoxic reaction in immunologytrial regimentrial treatmentvalidationsvector
Description preview
PROJECT SUMMARY
Over 7 million Americans suffer from Alzheimer’s Disease (AD), presenting a major health burden to patients
and their caregivers, and claiming over a hundred thousand lives annually. AD remains without any effective
cure or treatments, despite decades of research into small molecule- and antibody-based therapeutics. In
contrast,…
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