microRNA – A Small Biological Footprint with a Big Impact

A liver cell contains the same genomic information as an epidermal skin cell, yet these cell types have profoundly different function. If all cells are given the same set of instructions via chromosomal DNA, how do these differences develop? The answer lies in the selective expression and then regulation of certain genes in certain cell types. On December 10, 2024, Victor Ambros and Gary Ruvkun gave the 2024 Nobel Prize lecture in Physiology or Medicine, awarded for their “discovery of microRNA and its role in post-transcriptional gene regulation”. MicroRNAs, or miRNAs, are non-coding RNA molecules that pair with and selectively silence messenger RNA (mRNA), controlling gene expression and therefore downstream protein synthesis. They were completely unknown to science until Ambros and Ruvkun identified this radically new mechanism of gene regulation in the roundworm C. elegans, soon showing that it was universal across the animal kingdom.

Our growing ability to manipulate gene regulation has been the defining story of biology in the 21st century, making this the Century of Biotechnology. CRISPR-Cas9 and related technologies have enabled precise genome editing, opening doors to treating genetic disorders, engineering disease-resistant crops, and advancing synthetic biology. Epigenetic therapies targeting DNA methylation and histone modification are transforming cancer treatment and regenerative medicine. Single-cell RNA sequencing helps us unravel complex gene networks, shedding light on developmental biology and immune responses.

This year’s Nobel Prize in Physiology or Medicine is for recognition for two groundbreaking companion papers first published in 1993. In the thirty years since then, there’s been a flowering of miRNA research, from theory to practice. MicroRNA dysfunction has been implicated in a host of diseases, from cancers to addiction to heart disease, enabling new therapies and advancing the constant human struggle for a better world. Wherever basic science goes, biotechnology – and Georgetown Biotech – follow, drawing real-world applications from revolutions in the lab. Congratulations to Drs. Ambros and Ruvkun on recognition for their painstaking and transformative work.

Nobel acceptance speech- Ambros-Ruvkun, December 2024

Written by: Kyle DiVito, PhD

Georgetown University Biotechnology Program

December 2024