Restoration of Full-Length SMN Promoted by Adenoviral Vectors Expressing RNA Antisense Oligonucleotides Embedded in U7 snRNAs 英文参考文献.docVIP

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Restoration of Full-Length SMN Promoted by Adenoviral Vectors Expressing RNA Antisense Oligonucleotides Embedded in U7 snRNAs 英文参考文献.doc

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Restoration of Full-Length SMN Promoted by Adenoviral Vectors Expressing RNA Antisense Oligonucleotides Embedded in U7 snRNAs 英文参考文献

RestorationofFull-LengthSMNPromotedbyAdenoviral VectorsExpressingRNAAntisenseOligonucleotides EmbeddedinU7snRNAs TillGeib,KlemensJ.Hertel* DepartmentofMicrobiologyandMolecularGenetics,SchoolofMedicine,UniversityofCaliforniaIrvine,Irvine,California,UnitedStatesofAmerica Abstract Background:SpinalMuscularAtrophy(SMA)isanautosomalrecessivediseasethatleadstospecificlossofmotorneurons. Itiscausedbydeletionsormutationsofthesurvivalofmotorneuron1gene(SMN1).Theremainingcopyofthegene,SMN2, generatesonlylowlevelsoftheSMNproteinduetoamutationinSMN2exon7thatleadstoexonskipping. Methodology/PrincipalFindings:TocorrectSMN2splicing,weuseAdenovirustype5–derivedvectorstoexpressSMN2- antisenseU7snRNAoligonucleotidestargetingtheSMNintron7/exon8junction.InfectionofSMAtypeI–derivedpatient fibroblastswiththesevectorsresultedinincreasedlevelsofexon7inclusion,upregulatingtheexpressionofSMNtosimilar levelsasinnon–SMAcontrolcells. Conclusions/Significance: These results show that Adenovirus type 5–derived vectors delivering U7 antisense oligonucleotidescanefficientlyrestorefull-lengthSMNproteinandsuggestthattheviralvector-mediatedoligonucleotide applicationmaybeasuitabletherapeuticapproachtocounteractSMA. Citation:GeibT,HertelKJ(2009)RestorationofFull-LengthSMNPromotedbyAdenoviralVectorsExpressingRNAAntisenseOligonucleotidesEmbeddedinU7 snRNAs.PLoSONE4(12):e8204.doi:10.1371/journal.pone.0008204 Editor:JuanValcarcel,CentredeRegulacio′ Geno`mica,Spain ReceivedSeptember13,2009;AcceptedNovember11,2009;PublishedDecember8,2009 Copyright: ? 2009 Geib, Hertel. This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricteduse,distribution,andreproductioninanymedium,providedtheoriginalauthorandsourcearecredited. Funding:ThisworkwassupportedbyNIHgrantGM62287(KJH),aMuscularDystrophygrantMDA4043(KJH),andaFamiliesofSMAgrantHER0607(KJH).TG wassupportedbyafellowshipfromtheDeutscheForschungsgemeinschaft.Thefundershadnoroleinstudydesig