Compared to the well-known therapies centered around double-stranded DNA, the development of RNA therapeutics have advanced rather slowly. Scientists discovered the existence of messenger RNA (mRNA) in the 1960s, but the application of RNA editing began late in the 1990s. It was not until recently, the past two decades to be precise, that the development of RNA-related vaccines and the markets of new drug discovery began to flourish.

Although the techniques of DNA editing have been established longer, the major limitation lies in its irreversible characteristics; once DNA editing occurs, any unintended errors in the genome can’t be repaired easily. In contrast, RNA editing is reversible and produces fewer adverse reactions since RNAs are transient molecules. In this special series, we will focus on this potential gene-editing tool, introducing its history, clinical applications, industry trends, and interviews with experts in the field.

Discovery of RNA Editing in Living Organisms

The discovery of RNA editing can be traced back to 1986. Biologist Rob Benne and his group published a paper in the journal Cell focusing on the RNA editing mechanism in protozoa, marking the first discovery of RNA editing in living organisms. They observed that the transcript of the cytochrome oxidase coxII gene in the protozoan mitochondria has four inserted Uracil pairs not encoded by DNA. They named it “ RNA editing,” which refers to the phenomenon of post-transcriptional modifications on the RNA.