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 GrantFY2025AAV deliveredAAV deliveryAAV-based deliveryAAV-based viral deliveryAAV-mediated deliveryAccelerationAddressAdeno-associated-virus-based deliveryAdmissionAdmission activityAffectAmmoniaBiochemical PathwayBiodistributionBirthBloodBlood Reticuloendothelial SystemBrainBrain 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 technologyCarbamoyl PhosphateCarbamyl PhosphateCas nuclease technologyCatabolismCessation of lifeClinicClinicalClinical 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 mutationDataDeathDevelopmentDiagnosisDiseaseDisorderDoseDrugsEmergenciesEmergency SituationEncephalonEnsureEnzyme GeneEnzymesEvaluationFeedbackFood and Drug AdministrationFundingGene variantGenesGenetic ChangeGenetic defectGenetic mutationGuide RNAHealthHepaticHepatic TransplantationHereditary DiseaseHereditary Metabolic DisorderHeterozygoteHumanIND FilingIND applicationIND packageIND submissionImpairmentIn VitroInborn Errors of MetabolismInborn Genetic DiseasesInfantInherited disorderInvestigational DrugsInvestigational New Drug ApplicationInvestigational New DrugsLabelLearningLethargiesLifeLigaseLigase GeneLiverLiver GraftingLiver TransplantMedicalMedicationMetabolicMetabolic ControlMetabolic DiseasesMetabolic DisorderMetabolic NetworksMiceMice MammalsModern ManMorbidityMorbidity - disease rateMurineMusMutationNeonatalNeonatal ScreeningNervous System InjuriesNervous System TraumaNervous System damageNeurologicNeurologicalNeurological DamageNeurological InjuryNeurological traumaNewborn Infant ScreeningOrganParturitionPathogenesisPathogenicityPatientsPeer ReviewPharmaceutical PreparationsPhenylalaninePhenylalanine hydroxylase deficiencyPhenylketonuriasPlayPre IND FDA meetingPre-IND mtgProtocolProtocols documentationRNA SequencesResearchRespiratory distressRiskRoleSafetyScheduleSynthetasesSystemTherapeuticTherapeutic Gene EditingThesaurismosisTimeToxicologyTransplantationUSFDAUnited States Food and Drug AdministrationUrea cycle disordersVariantVariationWorkadeno-associated viral vector deliveryadeno-associated virus deliveryadeno-associated virus mediated deliveryadenovirus mediated deliveryallelic variantautosomebiomedical referral centercell transductioncellular transductioncohortdelivered with AAVdelivery with AAVdevelop therapydevelopmentaldrug/agentexperiencegRNAgene testinggene-based testinggene-editing therapygenetic diagnosisgenetic disorder diagnosisgenetic testinggenetic variantgenome editing based therapygenome editing therapygenome editing treatmentgenome editing-based therapeuticsgenome mutationgenomic variantgood laboratory practicehepatic body systemhepatic organ systemhepatoma cellhereditary disorderheritable disorderheterozygosityhigh riskimprovedimproved outcomein vivoinborn errorinborn metabolism disorderindividual patientinherited diseasesinherited genetic diseaseinherited genetic disorderinnovateinnovationinnovativeinpatient careinpatient serviceintervention developmentliver transplantationmanufacturemetabolism disordermonoanhydride with phosphoric acid Carbamic acidmortalityneonateneurotoxicneurotraumanewborn screeningnon-human primatenonhuman primateorganic acidphenylalaninemiapost-transplantpost-transplantationposttransplantposttransplantationpre-IND consultationpre-IND discussionpre-IND meetingpre-Investigational New Drug meetingpre-clinicalpre-clinical developmentpreclinicalpreclinical developmentprime editingprime editorprogramsreferral centersocial rolesomatic cell gene editingsomatic cell genome editingsomatic gene editingsomatic genome editingstandard of caretherapeutic editingtherapeutic genome editingtherapy developmenttransduced cellstransplanttreatment developmenttreatment strategy
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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