SignalTransductionandTargetedTherapywww.nature.com/sigtransREVIEWARTICLEOPENNucleicaciddrugs:recentprogressandfutureperspectivesXiaoyiSun1,SarraSetrerrahmane2,ChenchengLi1,JialiangHu1andHanmeiXu1✉Highefficacy,selectivityandcellulartargetingoftherapeuticagentshasbeenanactiveareaofinvestigationfordecades.Currently,mostclinicallyapprovedtherapeuticsaresmallmoleculesorprotein/antibodybiologics.Targetedactionofsmallmoleculedrugsremainsachallengeinmedicine.Inaddition,manydiseasesareconsidered‘undruggable’usingstandardbiomacromolecules.Manyofthesechallengeshowever,canbeaddressedusingnucleictherapeutics.Nucleicaciddrugs(NADs)areanewgenerationofgene-editingmoda...
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