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Journal Article
mRNA-based gene delivery shows promise
2012
Overview
The use of mRNA-based gene therapy avoids many of the side effects and risks associated with DNA-based plasmid and viral gene vectors. However, in vitro transcribed RNA activates cellular RNA sensors, leading to degradation and poor expression. This problem can be avoided in cultured cells by using highly-purified mRNAs containing the modified nucleoside pseudouridine. Kariko et al. show that a single injection of such modified mRNA encoding erythropoietin (EPO) elevates serum EPO levels in both mice and macaques, suggesting the possible clinical utility of modified mRNAs. Another paper, by Al-Saif and Khabar, shows that UU/UA dinucleotide frequency reduction in the coding region also enhances mRNA stability and protein expression.
Subject
