grant
Computational tools for precision genome editing
Organization BOSTON CHILDREN'S HOSPITALLocation BOSTON, UNITED STATESPosted 15 Jul 2024Deadline 30 Apr 2028
NIHUS FederalResearch GrantFY2025AddressAfrican ancestryAfrican descentAllelesAllelomorphsArtifactsAssayAutoregulationB-thalassemiaBasic ResearchBasic ScienceBenchmarkingBest Practice AnalysisBioassayBiological AssayBlind SpotsCRISPRCRISPR approachCRISPR based approachCRISPR based therapeuticsCRISPR based treatmentCRISPR methodCRISPR methodologyCRISPR techniqueCRISPR technologyCRISPR therapeuticsCRISPR toolsCRISPR treatmentCRISPR-CAS-9CRISPR-Cas based therapeuticsCRISPR-based disease therapeuticsCRISPR-based methodCRISPR-based techniqueCRISPR-based technologyCRISPR-based therapyCRISPR-based toolCRISPR/CAS approachCRISPR/Cas methodCRISPR/Cas systemCRISPR/Cas technologyCRISPR/Cas therapeuticsCRISPR/Cas9CRISPR/Cas9 technologyCRISPR/Cas9 therapeuticsCRISPR/Cas9 therapyCRISPR/Cas9 treatmentCRISPR/Cas9-based therapyCas nuclease technologyCas9 based therapeuticsCell BodyCell LineCellLineCellsClustered Regularly Interspaced Short Palindromic RepeatsClustered Regularly Interspaced Short Palindromic Repeats approachClustered Regularly Interspaced Short Palindromic Repeats based therapeuticsClustered Regularly Interspaced Short Palindromic Repeats methodClustered Regularly Interspaced Short Palindromic Repeats methodologyClustered Regularly Interspaced Short Palindromic Repeats techniqueClustered Regularly Interspaced Short Palindromic Repeats technologyClustered Regularly Interspaced Short Palindromic Repeats therapeuticsComplexComputational toolkitComputer softwareComputing MethodologiesDNADNA SequenceDNA seqDNA sequencingDNAseqData SetDeoxyribonucleic AcidDevelopmentDiseaseDisorderEnsureEnzyme GeneEnzymesEquityEventExperimental DesignsGene variantGenesGenomeGenomicsGenotypeGoalsGraphHb SS diseaseHbSS diseaseHemoglobin S DiseaseHemoglobin sickle cell diseaseHemoglobin sickle cell disorderHeritabilityHeterogeneityHeterozygoteHigh-Throughput Nucleotide SequencingHigh-Throughput SequencingHomeostasisHumanHuman GenomeIndividualInvestigatorsKnowledgeMediatingMethodologyMethodsModelingModern ManModificationMorphologic artifactsOutcomePathologyPathway interactionsPersonsPhysiological HomeostasisPopulationProbabilistic ModelsProbability ModelsReproducibilityReproducibility of FindingsReproducibility of ResultsResearchResearch PersonnelResearchersRetinal blind spotRisk AssessmentSafetyScientistSickle Cell AnemiaSiteSoftwareSpecificityStatistical Data AnalysesStatistical Data AnalysisStatistical Data InterpretationStatistical ModelsStrains Cell LinesTechnologyTestingTherapeuticTherapeutic Gene EditingTransposaseTreatment EfficacyVariantVariationWorkWritingallelic variantbase editingbenchmarkbeta Thalassemiaclinical applicabilityclinical applicationclinical developmentclinical relevanceclinical validationclinically relevantcomputational methodologycomputational methodscomputational toolboxcomputational toolscomputational toolsetcomputer based methodcomputer methodscomputerized toolscomputing methodcultured cell linedesigndesigningdevelopmentaldisease preventiondisorder preventionempowermentexperimentexperimental researchexperimental studyexperimentsflexibilityflexiblegene editing methodgene editing methodologygene editing strategygene editing techniquesgene-editing approachgene-editing therapygenetic variantgenome editinggenome editing based therapygenome editing therapygenome editing treatmentgenome editing-based therapeuticsgenome scalegenome-widegenomewidegenomic editinggenomic variantheterozygosityhuman whole genomeinnovateinnovationinnovativeintervention efficacylow-frequency mutationnovelnucleaseoff-target siteopen source toolopen source toolkitp-Thalassemiapan-genomepangenomepathwaypredictive toolspreventpreventingprime editingrare allelerare mutationrare variantrecombinasereference assemblyreference genomerepairrepairedsickle cell diseasesickle cell disordersickle diseasesicklemiasimulationstatistical analysisstatistical linear mixed modelsstatistical linear modelsstructural mutationstructural variantstructural variationtelomeretherapeutic agent developmenttherapeutic developmenttherapeutic editingtherapeutic efficacytherapeutic genome editingtherapeutically effectivetherapy efficacytoolβ-thalassemia
Description preview
PROJECT SUMMARY
This project proposes to develop and refine computational tools to advance the safety and efficacy of genome
editing, addressing the critical need for precise quantification and prediction of on- and off-target effects. Genome
editing technologies have the potential to revolutionize the treatment and prevention of disease by…
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