Treating Disease by Manipulating RNA

Our cells carry DNA, which encodes for all of the proteins that construct the body and carry out its functions. The cell uses RNA to carry information from the genome to cellular machinery that translates it into protein. Dysfunction in the genome can lead to disruptions in the structure of proteins and the processes they’re involved in, which can cause disease.

Researchers have long searched for ways to repair disease-causing errors in the human genome. However, tinkering with the genome can be dangerous and difficult. Some scientists have looked instead to RNA as a way to fix those problems. Learn more about various ways investigators are trying the approach from this video by the New England Journal of Medicine.