A broad spectrum of disease processes is caused by, or is associated with, abnormal protein levels.
Uncovering how protein expression is regulated in normal cells and how these processes can go wrong in disease, is therefore a crucial step to identify novel therapeutic options.
While academic research and the pharmaceutical industry initially focused on modifications of DNA, a growing body of evidence indicates that modifications of mRNA have a unique role in determining the degree to which genes are translated into proteins.
The Epitranscriptomics Machinery
In recent years, major discoveries in RNA-modification-mediated regulation of gene expression have been made, leading to the emerging field of epitranscriptomics.
The discovery of proteins that can add, remove and interpret these modifications – the writers, the erasers and the readers – has opened up a toolbox for drug development activities.
In 2012, our co-founder Samie Jaffray and his team advanced the concept of mRNA regulation by "epitranscriptomic" modification, i.e., the control of mRNA fate by chemical modifications of nucleotides in mRNA. They developed a technology to globally profile one chemically modified nucleotide demonstrating that chemically modified bases were widespread throughout the transcriptome, dynamically regulated during development and disease, and have regulatory potential. This work is often described as the study that launched the resurgence of interest in epitranscriptomics.