Nobel Prize for discovery of miRNAs!

Vera Mucaj
3 min readOct 9, 2024

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I look forward to Nobel Prize week like some people look forward to the NFL draft 😊.

This year has not disappointed! Monday’s announcement made my day, as it celebrated the scientists behind the discovery of the little engines that could… regulate gene expression! Micro-RNAs (or #miRNAs/miRs) are fascinating. Unlike messenger RNAs, miRNAs are “noncoding” as in, they don’t help produce proteins. Instead, they’re responsible for large-scale down-regulation of protein expression, across many targets. In doing so, they can control organ development, cell fate, tumorigenesis, and more. Better understanding miRNA biology can help us elucidate biological processes and even develop new drug treatment modalities! Congratulations to Drs Victor Ambros and Gary Ruvkun on a very well-deserved recognition!

(Fun fact: my labmate and I met Dr. Ambros at a Keystone microRNA conference back in ~2011. He sat with us at dinner and regaled us with small talk and scientific knowledge. He seemed like a genuinely kind person and brilliant scientist. We were starstruck, and I’ve been secretly rooting for him to receive the Nobel since!)

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Which one’s your favorite miRNA, Vera? Glad you asked 😊! It’s miR-124. I liked it so much that I did my PhD on it! Here’s three ways in which miR-124 is awesome:

1. It promotes neuronal differentiation by “turning off” genes that keep cells in an undifferentiated non-neuronal state. This is important because it ensures proper brain development, and allows neurons to form the connections needed for thinking, learning, and, well, living. This process was discovered by Dr. Eugene Makeyev back when he was a postdoctoral fellow in Dr. Tom Maniatis’ lab at Harvard.

2. It prevents cell survival under nutrient and oxygen deprivation. Wait, when would you want that? For starters, in cancer. A better way to think about it is to see what happens when levels of miR-124 are low. This is the case in brain tumors like the deadly glioblastoma multiforme (GBM). These tumors are notorious for being able to survive (and even be more aggressive) under low nutrients and hypoxia, which makes them very difficult to treat. Using miR-124 as a therapeutic agent in GBM targets those pro-survival signals and kills even the most difficult to target cells. This was the work I did with the brilliant Dr. Lijoy Mathew in Dr. Celeste Simon’s lab at the University of Pennsylvania. I hope some day these findings will result in new therapeutic approaches against this vicious tumor.

3. It can turn skin cells into neurons… with a little help from a friend. As shown by Dr. Andrew Yoo and the Crabtree lab, a concoction of miR-124 and miR-9/9* was shown to be able to turn human fibroblasts into functional dopaminergic neurons. There’s a lot of reasons why this is interesting, but primarily because cellular trans-differentiation (the process of going from one fully differentiated cell to another fully differentiated cell, without a stem cell being involved) is hard. This, to me, is still science-fiction-wild! It opens up immense possibilities in regenerative medicine. If we can make neurons out of skin, what else can we make?

While you might not end up picking a favorite microRNA any day soon, I hope you enjoyed learning a bit about this field!

Image sourced here

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Vera Mucaj
Vera Mucaj

Written by Vera Mucaj

Passionate about R&D and healthcare data. For more science-focused ideas, please visit my blog "Thought Experiments" at https://veramucaj.substack.com

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