Spliceosome-Mediated Trans-Splicing: The Therapeutic Cut and Paste

• Published in: Journal of investigative dermatology Vol. 132; no. 8; pp. 1959 - 1966
• Main Authors: Wally, Verena, Murauer, Eva M., Bauer, Johann W.
• Format: Journal Article
• Language: English
• Published: New York, NY Elsevier Inc 01.08.2012; Nature Publishing Group; Elsevier Limited
• Subjects: Alleles; Animals; Biological and medical sciences; Carcinoma, Squamous Cell - genetics; Cystic Fibrosis Transmembrane Conductance Regulator - genetics; Dermatology; Epidermolysis bullosa; Genetic Diseases, Inborn - genetics; Genetic Techniques; Genetic Therapy - methods; Genetic Therapy - trends; Humans; Medical sciences; Mice; Models, Genetic; Mutation; Neurodegenerative diseases; Phenotype; Reviews; RNA; RNA Splicing; Spliceosomes - genetics; Spliceosomes - metabolism; Therapeutic applications; Trans-Splicing; Splicing; Dermatology; Treatment
• ISSN: 0022-202X; 1523-1747; 1523-1747
• DOI: 10.1038/jid.2012.101

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.