title: RNA reprogramming and repair based on trans-splicing group I ribozymes.
abstract: While many traditional gene therapy strategies attempt to deliver new copies of wild-type genes back to cells harboring the defective genes, RNA-directed strategies offer a range of novel therapeutic applications. Revision or reprogramming of mRNA is a form of gene therapy that modifies mRNA without directly changing the transcriptional regulation or the genomic gene sequence. Group I ribozymes can be engineered to act in trans by recognizing a separate RNA molecule in a sequence-specific manner, and to covalently link a new RNA sequence to this separate RNA molecule. Group I ribozymes have been shown to repair defective transcripts that cause human genetic or malignant diseases, as well as to replace transcript sequences by foreign RNA resulting in new cellular functions. This review provides an overview of current strategies using trans-splicing group I ribozymes in RNA repair and reprogramming.
authors.affiliation: RNA and Transcriptomics Group, Department of Medical Biology, Faculty of Health Sciences, University of Tromsø, Tromsø, Norway. tonje.fiskaa@uit.no
authors.forename: Tonje
authors.initials: T
authors.lastname: Fiskaa
authors.forename: Asa B
authors.initials: AB
authors.lastname: Birgisdottir
mesh_terms: D000818:Animals; D001483:Base Sequence; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D007438:Introns; D008969:Molecular Sequence Data; D009690:Nucleic Acid Conformation; D012313:RNA / Q000235:genetics* / Q000378:metabolism*; D016337:RNA, Catalytic / Q000737:chemistry / Q000235:genetics / Q000378:metabolism*; D020040:Trans-Splicing
chemical_list: C116167:GIR1 ribozyme; D016337:RNA, Catalytic; D012313:RNA