precision medicine

Tall or short? Blue irises or brown? Human genetic material, or deoxyribonucleic acid (DNA), include genes that provide cellular machinery with instructions to produce functional proteins. These proteins ultimately determine many traits and support our daily activities. However, due to cell aging or environmental exposure to chemicals or radiation, mutations can happen randomly within trait-encoding genes, leading to problematic gene expression and protein activity (Figure 1).

In genetics, it is common knowledge that the blueprint of life lies within the intricate structure of DNA. However, a lesser-known but equally important player in the process of gene expression is RNA. From my previous blog, we learned that RNA carries the instructions encoded in DNA and helps to synthesize proteins that dictate the functioning of living organisms.

The development of mRNA vaccines against COVID-19 has showcased the potential of RNA-based approaches, generating significant enthusiasm and investment in the field. RNA-based therapeutics offer new potential treatments for a range of diseases, taking advantage of the natural role of RNA in gene expression and allowing for targeted gene regulation and protein synthesis. In this blog, we will explore the types of RNA-based therapeutics, their advantages and limitations, and the current state of research in the field. But first, let’s define what an RNA molecule is.