title: Spliceosome-mediated trans-splicing: the therapeutic cut and paste.
abstract: Spliceosome-mediated RNA trans-splicing (SMaRT) is an RNA-based technology to reprogram genes for diagnostic and therapeutic purposes. For the correction of genetic diseases, SMaRT offers several advantages over traditional gene-replacement strategies. SMaRT protocols have recently been used for in vitro phenotypic correction of a variety of genetic disorders, ranging from epidermolysis bullosa to neurodegenerative diseases. In vivo studies are currently bringing trans-splicing RNA therapy toward clinical application. In this review, we summarize the progress made toward the medical use of SMaRT and provide an outlook on its upcoming applications.
authors.affiliation: Division of Molecular Dermatology and EB House Austria, Department of Dermatology, Paracelsus Medical University, Salzburg, Austria.
authors.forename: Verena
authors.initials: V
authors.lastname: Wally
authors.forename: Eva M
authors.initials: EM
authors.lastname: Murauer
authors.forename: Johann W
authors.initials: JW
authors.lastname: Bauer
mesh_terms: D000483:Alleles; D000818:Animals; D002294:Carcinoma, Squamous Cell / Q000235:genetics; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics; D030342:Genetic Diseases, Inborn / Q000235:genetics*; D005821:Genetic Techniques; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D006801:Humans; D051379:Mice; D008957:Models, Genetic; D009154:Mutation; D010641:Phenotype; D012326:RNA Splicing; D017381:Spliceosomes / Q000235:genetics* / Q000378:metabolism; D020040:Trans-Splicing
chemical_list: C505032:CFTR protein, human; D019005:Cystic Fibrosis Transmembrane Conductance Regulator