title: The notorious R.N.A. in the spotlight - drug or target for the treatment of disease.
abstract: mRNA is an attractive drug target for therapeutic interventions. In this review we highlight the current state, clinical trials, and developments in antisense therapy, including the classical approaches like RNaseH-dependent oligomers, splice-switching oligomers, aptamers, and therapeutic RNA interference. Furthermore, we provide an overview on emerging concepts for using RNA in therapeutic settings including protein replacement by in-vitro-transcribed mRNAs, mRNA as vaccines and anti-allergic drugs. Finally, we give a brief outlook on early-stage RNA repair approaches that apply endogenous or engineered proteins in combination with short RNAs or chemically stabilized oligomers for the re-programming of point mutations, RNA modifications, and frame shift mutations directly on the endogenous mRNA.
authors.affiliation: a Interfaculty Institute of Biochemistry, University of Tübingen Auf der Morgenstelle , Tübingen , Germany.
authors.forename: Philipp
authors.initials: P
authors.lastname: Reautschnig
authors.affiliation: a Interfaculty Institute of Biochemistry, University of Tübingen Auf der Morgenstelle , Tübingen , Germany.
authors.forename: Paul
authors.initials: P
authors.lastname: Vogel
authors.affiliation: a Interfaculty Institute of Biochemistry, University of Tübingen Auf der Morgenstelle , Tübingen , Germany.
authors.forename: Thorsten
authors.initials: T
authors.lastname: Stafforst
mesh_terms: D052157:Aptamers, Nucleotide / Q000737:chemistry / Q000378:metabolism; D002986:Clinical Trials as Topic; D055808:Drug Discovery; D030342:Genetic Diseases, Inborn / Q000628:therapy*; D006801:Humans; D016376:Oligonucleotides, Antisense / Q000627:therapeutic use; D017393:RNA Editing; D034622:RNA Interference; D020871:RNA Stability; D016372:RNA, Antisense / Q000627:therapeutic use*; D012333:RNA, Messenger / Q000235:genetics / Q000378:metabolism; D000067492:RNAi Therapeutics; D016914:Ribonuclease H / Q000378:metabolism*
chemical_list: D052157:Aptamers, Nucleotide; D016376:Oligonucleotides, Antisense; D016372:RNA, Antisense; D012333:RNA, Messenger; D016914:Ribonuclease H
keywords: Antisense oligonucleotide; RNA interference; RNA repair; RNA vaccines; chemically modified oligonucleotides; genetic disease; site-directed RNA editing; splice-switching oligonucleotide; therapeutic aptamer; therapeutic mRNA