Article
Design and characterization of a twin ribozyme for potential repair of a deletion mutation within the oncogenic CTNNB1-ΔS45 mRNA.
ChemMedChem - 1 Sept 2014
Balke Darko, Zieten Irene, Strahl Anne, Müller Oliver, Müller Sabine
Abstract excerpt
RNA repair is an emerging strategy for gene therapy. Conventional gene therapy typically relies on the addition of the corrected DNA sequence of a defective gene to restore gene function. As an additional option, RNA repair allows alteration of the sequence of endogenous messenger RNAs (mRNAs). mRNA sequence alteration is either facilitated by intracellular spliceosome machinery or by the intrinsic catalytic...
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