grant
High Fidelity Genome Editing for the Correction of MECP2 Mutations and Physiologic Regulation of Expression in Rett Syndrome
Organization BECKMAN RESEARCH INSTITUTE/CITY OF HOPELocation DUARTE, UNITED STATESPosted 15 Apr 2025Deadline 28 Feb 2030
NIHUS FederalResearch GrantFY2025AddressAffectAvesAvianBBB crossingBirdsBody TissuesBrainBrain Nervous SystemCNS Nervous SystemCell BodyCell Communication and SignalingCell CycleCell Division CycleCell SignalingCellsCentral Nervous SystemCerebroatrophic HyperammonemiaChromatin Remodeling ComplexChromatin Remodeling FactorClinicalCodeCoding SystemCognitiveCoupledDNA TherapyDNA mutationDataDegenerative Neurologic DisordersDiseaseDisease ProgressionDisorderEncephalonEpigeneticEpigenetic ChangeEpigenetic MechanismEpigenetic ProcessEvaluationExonsFemaleFrequenciesGene AlterationGene MutationGene TranscriptionGene Transfer ClinicalGeneralized GrowthGenesGeneticGenetic ChangeGenetic DiseasesGenetic InterventionGenetic TranscriptionGenetic defectGenetic mutationGenetics-MutagenesisGenomeGoalsGrowthHeterozygoteHumanIn VitroIncrease lifespanInduced pluripotent stem cell derived neuronsIntracellular Communication and SignalingIntravenousKnock-outKnockoutKnowledgeLanguageLifeLinkLiverMeCP-2 proteinMeCP2MeCP2 proteinMediatingMessenger RNAMethyl CpG binding protein MeCP2Methyl-CpG-Binding Protein 2Methyl-DNA binding protein MECP2MiceMice MammalsMissense MutationModelingModern ManModificationMolecularMurineMusMutagenesisMutagenesis Molecular BiologyMutationNerve CellsNerve UnitNervous System Degenerative DiseasesNeural CellNeural Degenerative DiseasesNeural degenerative DisordersNeuraxisNeurocyteNeurodegenerative DiseasesNeurodegenerative DisordersNeurodevelopmental DisorderNeurologic Degenerative ConditionsNeurological Development DisorderNeuron from iPSCNeuron from induced pluripotent stem cellsNeuronsOrganoidsOther GeneticsPathogenesisPathogenicityPatientsPeripheralPhenotypePhysiologicPhysiologicalPreclinical dataProblem behaviorProgenitor CellsProteinsRNA ExpressionReaderRegulationRegulatory ElementRett DisorderRett SyndromeRiskSafetySeizuresSerotypingSignal TransductionSignal Transduction SystemsSignalingSymptomsTestingTherapeuticTherapeutic EffectTimeTissue GrowthTissuesToxic effectToxicitiesTranscriptionTropismVirusalleviate symptomameliorating symptombehavioral problembiological signal transductionblood-brain barrier crossingbloodbrain barrier crossingboost longevitychromatin modifierde novo mutationde novo variantdecrease symptomdeep sequencingdegenerative diseases of motor and sensory neuronsdegenerative neurological diseaseselongating the lifespanenhance longevityepigeneticallyextend life spanextend lifespanextend longevityfewer symptomsfoster longevityfunctional restorationgene defectgene editing methodgene editing methodologygene editing strategygene editing techniquesgene repair therapygene therapygene-based therapygene-editing approachgenetic conditiongenetic disordergenetic therapygenome editinggenome mutationgenome scalegenome-widegenomewidegenomic editinggenomic therapygenotoxicityhepatic body systemhepatic organ systemheterozygosityhomologous recombinationiPS neuronsiPSC derived-neuronsimprove lifespanimprove longevityimprovedin vivoinduced pluripotent stem cell neuronsintravenous injectionlanguage abilitylanguage skillslifespan extensionmRNAmalemissense single nucleotide polymorphismmissense single nucleotide variantmissense variantmouse modelmurine modelmutantmutant alleleneurodegenerative illnessneurodevelopmental diseaseneuronalneurons derived from induced pluripotent stem cellsneurons differentiated from induced pluripotent stem cellsnovelnucleaseontogenypostmitoticpreclinical findingspreclinical informationpreservationprolong lifespanprolong longevitypromote lifespanpromote longevityreduce symptomsrelieves symptomsrepairrepairedrestorationrestore functionrestore functionalityrestore lost functionstem cellssuccesssupport longevitysymptom alleviationsymptom reductionsymptom relieftrendvectorvector genome♀♂
Description preview
ABSTRACT
Rett syndrome (RTT) is an acquired progressively debilitating neurodevelopmental disorder caused by de novo
mutations in the X-linked MECP2 gene that is almost exclusively observed in heterozygous females while
hemizygous mutant males rarely survive. RTT is characterized by reduced brain growth, loss of mobility,
language skills,…
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