Gene Expression from Pseudourine and 5-Methylcytidine Modified Messenger RNA

Recently, there has been significant interest in the use of mRNA based expression systems for gene therapy applications and for the generation and manipulation of stem cells. Several groups have shown that mRNAs are attractive vehicles for therapeutic gene expression in mammals (Kormann et. al. Nat. Biotechnol (2011) 29, 154; Kariko et. al. Molecular Therapy (2012) Epub ahead of print). Additionally, Warren et al. demonstrated highly efficient induced pluripotent stem cell (iPSCs) generation by transfection of mRNAs encoding reprogramming factors (Warren et al. Cell Stem Cell (2010) 7, 618). The authors suggested that iPSCs generated in this manner should be safer than iPSCs derived by plasmid transfection or viral transduction as there is no risk of insertional mutagenesis and subsequent oncogenesis.