grant
Gene Delivery Core
Organization CINCINNATI CHILDRENS HOSP MED CTRLocation CINCINNATI, UNITED STATESPosted 1 Aug 2021Deadline 31 Jul 2028
NIHUS FederalResearch GrantFY2025AAV vectorAAV-based vectorAccelerationAd vectorAdenoviral VectorAdenovirus VectorAssayBasic ResearchBasic ScienceBioassayBiologic PhenomenaBiologicalBiological AssayBiological PhenomenaBiologyBlood DiseasesCRISPRCRISPR approachCRISPR based approachCRISPR libraryCRISPR methodCRISPR methodologyCRISPR techniqueCRISPR technologyCRISPR toolsCRISPR-CAS-9CRISPR-based libraryCRISPR-based methodCRISPR-based techniqueCRISPR-based technologyCRISPR-based toolCRISPR/CAS approachCRISPR/Cas methodCRISPR/Cas systemCRISPR/Cas technologyCRISPR/Cas9CRISPR/Cas9 libraryCRISPR/Cas9 technologyCas nuclease technologyCell BodyCellsCellular biologyChildren's HospitalClinicClinicalClinical TrialsClustered Regularly Interspaced Short Palindromic RepeatsClustered Regularly Interspaced Short Palindromic Repeats approachClustered Regularly Interspaced Short Palindromic Repeats libraryClustered Regularly Interspaced Short Palindromic Repeats methodClustered Regularly Interspaced Short Palindromic Repeats methodologyClustered Regularly Interspaced Short Palindromic Repeats techniqueClustered Regularly Interspaced Short Palindromic Repeats technologyCollaborationsCommunitiesComplexConsultationsCore FacilityCyclic GMPDNADNA TherapyDNA VirusesDeoxyribonucleic AcidDevelopmentDevelopment and ResearchDevelopmental TherapeuticsDevelopmental Therapeutics ProgramDevelopmental TherapyDiseaseDisorderDysfunctionEnsureEvaluationFeasibility StudiesFoamy VirusFunctional disorderFunding AgencyFunding SourceFutureGammaretrovirusGene DeliveryGene ExpressionGene ModifiedGene TransferGene Transfer ClinicalGenesGenetic InterventionGenetic studyGoalsGuanosine Cyclic MonophosphateGuide RNAHematologic DiseasesHematological DiseaseHematological DisorderHematologyHigh Throughput AssayHumanHuman GenomeHuman ResourcesIndividualInfrastructureInstitutionInvestigationInvestigational DrugsInvestigational New DrugsInvestigatorsKnock-outKnockoutLaboratoriesLaboratory ResearchLentiviral VectorLentivirinaeLentivirusLentivirus VectorLibrariesMammalian Type C RetrovirusesManpowerMaterials TestingMedical centerMethodologyMiceMice MammalsModern ManMolecularMonitorMurineMusNon-Polyadenylated RNAPediatric HospitalsPhasePhysiopathologyPreparationProceduresProcessProductionQuality ControlR & DR&DR-Series Research ProjectsR01 MechanismR01 ProgramRNARNA Gene ProductsReproducibilityResearchResearch GrantsResearch PersonnelResearch Project GrantsResearch ProjectsResearch ResourcesResearchersResourcesRibonucleic AcidRoboticsSerotypingServicesSimiispumavirusSourceSpumavirusSystemTechnologyTestingTimeTrainingTranslatingTranslational ResearchTranslational ScienceTranslationsUltracentrifugationViral Vectoradeno vectoradeno-associated viral vectoradeno-associated virus vectoradenovectorbarrier to translationbench bed sidebench bedsidebench to bed sidebench to bedsidebench to clinicbench to clinical practicebiologicbiological researchblood disordercGMPcell biologyclinical relevanceclinically relevantconsultationcostdelivery vectordelivery vehicledesigndesigningdevelopmentalearly clinical trialearly phase clinical trialexperiencegRNAgene manipulationgene modificationgene repair therapygene therapeuticsgene therapygene-based therapeuticgene-based therapeuticsgene-based therapygenes therapeuticgenes therapeuticsgenetic manipulationgenetic therapygenetically engineered cellsgenetically manipulategenetically modifiedgenetically modified cellsgenetically perturbgenomic therapyhigh throughput screeninghuman whole genomeindustrial partnershipindustry partnerindustry partnershipinnovateinnovationinnovativeinterestknock-downknockdownknockout genemanufacturemouse genomenew technologynovelnovel technologiesobstacle to translationpathophysiologypersonnelpre-clinicalpre-clinical studypreclinicalpreclinical studypreparationsresearch and developmentscale upscreeningscreeningsshRNAshort hairpin RNAsmall hairpin RNAsmall moleculetherapeutic agent developmenttherapeutic developmenttherapeutic genetooltranslationtranslation barriertranslation obstacletranslation researchtranslational barriertranslational investigationtranslational obstacletranslational pipelinetranslational spectrumvectorγ-retrovirus
Description preview
Abstract
Investigation of the molecular and cellular pathophysiology for hematologic diseases requires rapid and
low-cost access to vector preparations, and access to large libraries of vectors and production of vectors
destined to high throughput screening. Further, the successful development and testing of novel gene
therapies requires access to…
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