grant
Robust verification of genetic variant-associated candidate off-target sites
Organization BOSTON CHILDREN'S HOSPITALLocation BOSTON, UNITED STATESPosted 19 May 2025Deadline 30 Apr 2028
NIHUS FederalResearch GrantFY2025AccelerationAccountingAdoptedAll of Us ProgramAll of Us Research ProgramAll of Us Research ProjectAoURPAssayBenchmarkingBest Practice AnalysisBioassayBioinformaticsBiological AssayBlind SpotsBody TissuesCNS Nervous SystemCancersCell BodyCell TherapyCellsCentral Nervous SystemChromatinCollaborationsCommunitiesComputational toolkitComputer Software ToolsComputer softwareDNA TherapyDataData SetDiseaseDisorderEnsureEvaluationEye Nervous SystemFDA approvedFosteringFundingGene Transfer ClinicalGene variantGenesGenetic DiseasesGenetic DiversityGenetic InterventionGenetic VariationGenomeGenomicsGuide RNAHumanHuman GeneticsIND FilingIND applicationIND packageIND submissionIndividualIndustryInvestigational New Drug ApplicationInvestigatorsLeadLibrariesLiverMalignant NeoplasmsMalignant TumorMethodologyMethodsModalityModelingModern ManNeuraxisNeuro-Ocular SystemNoiseOutcomePb elementPerformancePlasmidsPopulationPositionPositioning AttributeProbabilistic ModelsProbability ModelsProceduresProcessProtocolProtocols documentationReportingResearch PersonnelResearchersRetinal blind spotRiskRisk AssessmentSafetySiteSoftwareSoftware ToolsSomatic MutationSpecificityStandardizationStatistical ModelsTestingTherapeutic Gene EditingTissuesTranslationsVariantVariationWorkallelic variantanalysis pipelinebase editorbenchmarkcell based interventioncell mediated interventioncell mediated therapiescell typecell-based therapeuticcell-based therapycellular therapeuticcellular therapyclinical developmentclinical relevanceclinical translationclinically relevantclinically translatablecomputational toolboxcomputational toolscomputational toolsetcomputerized toolscost effectivedeep sequencingdesigndesigningdetection limitdevelop softwaredeveloping computer softwaregRNAgene repair therapygene therapygene-based therapygene-editing therapygenetic conditiongenetic disordergenetic elementgenetic therapygenetic variantgenome editinggenome editing based therapygenome editing therapygenome editing treatmentgenome editing-based therapeuticsgenomic editinggenomic therapygenomic variantheavy metal Pbheavy metal leadhepatic body systemhepatic organ systemhigh standardhuman reference assemblyhuman reference genomeimprovedin silicoin vivoindividual patientmalignancymeetingmeetingsmemberneoplasm/cancernucleaseoff-target sitepatient populationpost-marketpre-clinical evaluationpreclinical evaluationpredictive toolsprime editorreference assemblyreference genomesimulationsoftware developmentsoftware toolkitsomatic variantstatistical linear mixed modelsstatistical linear modelssurveillance studytherapeutic editingtherapeutic genome editingtooltranslationuser friendly computer softwareuser friendly softwarevector
Description preview
PROJECT SUMMARY
Therapeutic genome editing holds immense promise for treating genetic diseases, but off-target effects remain
a critical safety concern, particularly when considering human genetic diversity. Most existing methods for
identifying off-target sites do not account for genetic variants, potentially missing important risks for…
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