Because RNAs are so central to our biology, they are also drivers of a wide array of diseases. Defects in how RNA is produced and processed can lead to cancer, degenerative diseases, metabolic disorders and autoimmune diseases. RNA viruses, such as HIV, influenza or SARSCoV2, also hijack our cellular machinery to cause disease.

“When the underlying machinery is not functioning in our cells, the problems that result are huge,” said Yeo. “But this also means we have a lot of opportunities to leverage RNAs to treat diseases and support human health.”

Putting RNAs to Work

According to many researchers, we are currently in the midst of a RNA revolution, with scientists around the world turning their gaze to these tiny molecules for disease diagnosis and treatment.

One disease Yeo has made strides in understanding and treating is myotonic muscular dystrophy type 1, a rare genetic disorder that slowly degrades muscles and other organs. One of the drivers of this disease is the expression of “toxic” RNA molecules within cells, but Yeo and his group discovered that these toxic RNAs can be eliminated using RNA-targeting gene therapies.

This approach could, according to Yeo, not only lead to cures for this particular disease, but could also be applicable to a wide array of diseases affecting millions of people around the world. That said, it will take more research to fully unlock this potential.