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Gene Therapy With Self-Complementary

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MolecularTherapyVolume23,Supplement1,May2015Copyright©TheAmericanSocietyofGene&CellTherapyS54GeneTarGeTinGandGeneCorreCTionimutationandcorrectionofSMN2aberrantsplicing,byexploitingthenon-homologousend-joining(NHEJ)pathway.PlasmidsencodingCas9-GFPunderthecontrolofCMVpromoter,andselectedgRNAsdownstreamtothePol-IIIU6promoter(Addgene)weretransfectedinHEK-293TcelllineandinimmortalizedmyoblastsderivedfromeitherhealthydonorsorSMApatients.TransfectionefficiencywasestimatedaspercentageofGFP-expressingcells(20-50%and1-10%,respectively)andnucleaseactivitydetectedbySurveyorassayandtargetsitesequencing.Inparticular,inSMApatient-derivedmyoblastswedetectedmutations(indels)attheleveloftheinducedDNAdouble-strandbreakat~30%frequency.LevelsofSMNrestorationwillbeinvestigatedbyqPCRofthedifferentspeciesofSMNtranscriptsandbywesternblottingofSMNprotein.ThegoalofthisstudyistoprovideaninvitroproofofprincipleofeffectivegenecorrectioninSMApatient-derivedcells.Inthecontextofamultisystemic,complicateddiseasesuchasSMA,targetedgenomeeditingstrategycouldrepresentanadditionaltherapeutictool.132.GeneTherapyWithSelf-ComplementaryRecombinantAdeno-AssociatedVirusinModelsofAutosomalDominantRetinitisPigmentosaCausebyRHOMutationsBrianRossmiller,1DannyZakria,1ArathiNandyala,1HiralJivanji,1LewinAlfred.11MolecularGeneticsandMicrobiology,TheUniversityofFlorida,Gainesville,FL.PurposeRetinitispigmentosaistheleadinghereditarycauseofblindnesswith30-40%ofcasesattributabletoautosomaldominantretinitispigmentosa(ADRP).ADRP

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