grant
Personalized prime editing as a platform for hepatic inborn errors of metabolism
Organization CHILDREN'S HOSP OF PHILADELPHIALocation PHILADELPHIA, UNITED STATESPosted 20 Sept 2024Deadline 31 Aug 2028
NIHUS FederalResearch GrantFY202521+ years oldAAV deliveredAAV deliveryAAV-based deliveryAAV-based viral deliveryAAV-mediated deliveryASA deficiencyAccelerationAccountingAddressAdeno-Associated VirusesAdeno-associated-virus-based deliveryAdultAdult HumanAffectAmmoniaArgininosuccinate Synthase Deficiency DiseaseArgininosuccinic Acid Synthase Deficiency DiseaseArgininosuccinic Acid Synthetase Deficiency DiseaseBiochemical PathwayBirthBrainBrain Nervous SystemCRISPR approachCRISPR based approachCRISPR methodCRISPR methodologyCRISPR techniqueCRISPR technologyCRISPR toolsCRISPR-CAS-9CRISPR-based methodCRISPR-based techniqueCRISPR-based technologyCRISPR-based toolCRISPR/CAS approachCRISPR/Cas methodCRISPR/Cas technologyCRISPR/Cas9CRISPR/Cas9 technologyCas nuclease technologyCatabolismCitrullinemiaCitrullinuriaClinicalClinical TrialsClustered Regularly Interspaced Short Palindromic Repeats approachClustered Regularly Interspaced Short Palindromic Repeats methodClustered Regularly Interspaced Short Palindromic Repeats methodologyClustered Regularly Interspaced Short Palindromic Repeats techniqueClustered Regularly Interspaced Short Palindromic Repeats technologyCommunitiesDNA mutationDependoparvovirusDependovirusDevelopmentDiseaseDisorderDoseDrug KineticsEncephalonEnrollmentEnzyme GeneEnzymesEthicsGene variantGenesGenetic ChangeGenetic defectGenetic mutationGuide RNAHealthHepaticHepatic TransplantationHereditary DiseaseHereditary Metabolic DisorderIND FilingIND applicationIND packageIND submissionImpairmentInborn Errors of MetabolismInborn Genetic DiseasesIndividualInherited disorderInstitutionInterventionInterviewInvestigational New Drug ApplicationLeadLifeLiverLiver GraftingLiver TransplantMediatingMedicalMetabolicMetabolic ControlMetabolic DiseasesMetabolic DisorderMetabolic NetworksMiceMice MammalsMorbidityMorbidity - disease rateMurineMusMutationNeonatalNeonatal ScreeningNervous System InjuriesNervous System TraumaNervous System damageNeurological DamageNeurological InjuryNeurological traumaNewborn Infant ScreeningOrganOutcomeParturitionPathogenesisPathogenicityPatientsPb elementPersonalized medical approachPharmacokineticsPharmacology and ToxicologyPhenotypePhenylalaninePhenylalanine 4-HydroxylasePhenylalanine 4-MonooxygenasePhenylalanine HydroxylasePhenylalanine hydroxylase deficiencyPhenylketonuriasPlayPre IND FDA meetingPre-IND mtgProtocolProtocols documentationRNA SequencesRiskRoleSafetyScientistSeriesSystemTarget PopulationsTherapeuticTherapeutic Gene EditingThesaurismosisTimeTransplantationUrea cycle disordersVariantVariationadeno associated virus groupadeno-associated viral vector deliveryadeno-associated virus deliveryadeno-associated virus mediated deliveryadenovirus mediated deliveryadulthoodallelic variantarginosuccinate synthetase deficiencyautosomebiomedical referral centercohortcommunity engaged participatory researchcommunity engaged researchcommunity partnered researchcommunity-engaged studycommunity-partnered studydelivered with AAVdelivery with AAVdevelop therapydevelopmentalenrollethicalexperiencegRNAgene-editing therapygenetic variantgenome editing based therapygenome editing therapygenome editing treatmentgenome editing-based therapeuticsgenome mutationgenomic variantheavy metal Pbheavy metal leadhepatic body systemhepatic organ systemhereditary disorderheritable disorderimprovedimproved outcomein vivoinborn errorinborn metabolism disorderindividualized approachinherited diseasesinherited genetic diseaseinherited genetic disorderinnovateinnovationinnovativeintervention developmentliver transplantationmeetingmeetingsmetabolism disordermortalityneonateneurotoxicneurotraumanewborn screeningnon-human primatenonhuman primateorganic acidpersonalization of treatmentpersonalized approachpersonalized medicinepersonalized therapypersonalized treatmentpharmacologicphenylalaninasephenylalaninemiapost-transplantpost-transplantationposttransplantposttransplantationpre-IND consultationpre-IND discussionpre-IND meetingpre-Investigational New Drug meetingpre-clinical studyprecision approachpreclinical studyprime editingprime editorprogramsreferral centersocial roletailored approachtherapeutic editingtherapeutic genome editingtherapy developmenttransplanttreatment developmenttreatment strategyuptake
Description preview
PROJECT SUMMARY
Inborn errors of metabolism (IEMs) are rare, devasting disorders arising from pathogenic variants in genes
encoding enzymes of key biochemical pathways. The liver plays an important role in the pathogenesis of over
150 IEMs, often failing to metabolize a toxic metabolite that can injure secondary organs, such as brain. Liver…
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