grant
Beginning genetic interventions in infants (BEGIN)
Organization UNIVERSITY OF MIAMI SCHOOL OF MEDICINELocation CORAL GABLES, UNITED STATESPosted 3 Apr 2026Deadline 31 Mar 2030
NIHUS FederalResearch GrantFY20260-11 years old5 year old5 years of ageASO therapeuticsASO therapyASO treatmentAccelerationAddressAdmissionAdmission activityAffectAlgorithmsAntisense AgentAntisense Oligonucleotide TherapyAntisense OligonucleotidesAwarenessBindingBiological Specimen BanksBiological Substance BanksBloodBlood Reticuloendothelial SystemBody TissuesBostonCaringChildChild YouthChildhoodChildren (0-21)Children's HospitalClinicalCollaborationsCommunicationCountryDNADNA TherapyDNA mutationDataDecreased Muscle ToneDeoxyribonucleic AcidDevelopmentDiagnosisDiagnosticDiseaseDisease ProgressionDisorderDisparitiesDisparityDrug DeliveryDrug Delivery SystemsDrug TargetingEarly DiagnosisEarly InterventionEnrollmentExhibitsFDA licensed drugsFDA-approved agentsFDA-approved drugFDA-approved medicationsFDA-approved pharmaceuticalsFDA-approved therapeutic agentFamilyFamily ResearchFibroblastsFood and Drug Administration approved drugFood and Drug Administration approved medicationsFood and Drug Administration approved pharmaceuticalsFormulationFosteringFoundationsFundingFutureGene SplicingGene Transfer ClinicalGenesGeneticGenetic ChangeGenetic DiseasesGenetic DiversityGenetic InterventionGenetic VariationGenetic defectGenetic mutationGenomeGenomic medicineGenomicsGoalsHealth Care ProvidersHealth PersonnelHypomyotoniaHypotoniaIncentivesIndustryInfantInfant HealthInterventionInvestmentsLaboratoriesLarge-Scale SequencingLifeLinkMental HealthMental HygieneMethodsModalityModelingMolecular InteractionMorbidityMuscle HypotonyMuscle Tone PoorMuscle hypotoniaMuscular HypotoniaMutationNational Institutes of HealthNatureNeonatal Intensive Care UnitsNeurologicNeurologicalNewborn Intensive Care UnitsNon-Polyadenylated RNANon-Profit OrganizationsNonprofit OrganizationsNucleotidesOutcomePBMCParticipantPathogenicityPatientsPatternPediatric HospitalsPenetrationPeripheral Blood Mononuclear CellPhenotypePopulationPrecision therapeuticsProviderPsychological HealthQualifyingRNARNA Gene ProductsRNA SeqRNA SplicingRNA sequencingRNA targeting drugRNA targeting therapeuticsRNA-targeting therapyRNAseqResearch ResourcesResearch SpecimenResource DevelopmentResourcesRibonucleic AcidSamplingSeizuresServicesShort interfering RNASiteSmall Interfering RNASourceSpecialistSpecimenSpliced GenesSplicingSurvey InstrumentSurveysSystemTechnologyTestingTherapeuticTherapeutic AgentsTherapeutic InterventionTimeTissuesTranscriptTranslationsUncertaintyUnited States National Institutes of HealthVariantVariationWorkadvanced diseaseadvanced illnessage 5age 5 yearsanti-sense oligonucleotide druganti-sense oligonucleotide therapyanti-sense oligonucleotide treatmentanti-sense therapyantisense drugantisense oligoantisense oligonucleotide therapeuticantisense therapeuticsantisense therapybiobankbiological specimen repositorybiorepositorybiosample repositorybiospecimen bankbiospecimen repositoryclinical practicecohortcustomized therapycustomized treatmentdesigndesigningdevelopmentaldisease causing variantdisease-causing alleledisease-causing mutationdoubtdrug developmentearly detectioneffective interventioneffective therapyeffective treatmentenrollexome sequencingexome-seqexpectationfamily based researchfamily centered researchfamily focused researchfamily investigationfive year oldfive years of ageflexibilityflexiblegain of function mutationgene functiongene repair therapygene replacement therapygene therapygene-based therapygene-based treatmentgene-directed therapygene-targeted therapygene-targeted treatmentgenetic conditiongenetic diagnosisgenetic disordergenetic disorder diagnosisgenetic therapygenome editinggenome medicinegenome mutationgenome sequencinggenomic editinggenomic therapyglobal gene expressionglobal transcription profilehealth care personnelhealth care workerhealth providerhealth staffhealth workershealth workforcehealthcare employeeshealthcare staffhealthcare workforcein silicoindividualized medicineindividualized patient treatmentindividualized therapeuticindividualized therapeutic strategyindividualized therapyindividualized treatmentinnovateinnovationinnovativeintervention therapyinvestigate familykidsknock-downknockdownlymphoblastoid cell linemedical care providersmedical personnelmortalityneonatal ICUpathogenic allelepathogenic variantpatient specific therapiespatient specific treatmentpediatricpersonalization of treatmentpersonalized medicinepersonalized therapeuticpersonalized therapypersonalized treatmentpharmacologicprecision medicineprecision therapiesprecision treatmentprecision-based medicinepreservationrare genetic diseaserare genetic disordersatisfactionsiRNAspecimen bankspecimen repositorystudy familysurvey familysynthetic DNAsynthetic constructtailored medical treatmenttailored therapytailored treatmenttherapeutic agent developmenttherapeutic developmenttranscriptometranscriptome sequencingtranscriptomic sequencingtranslationtranslational genomicstreatment providerunique treatmentvirtualyoungster
Description preview
PROJECT SUMMARY
The advancements in genomic medicine have significantly accelerated over the past decade. However, their
translation into clinical practice have been slow, and the precise treatment options are essentially nonexistent.
The formulation of effective therapies is obstructed by the vast diversity of genetic diseases and the limited…
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