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The MicroRNA Perspective

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Andrea Ventura
Title: Assistant Member, Memorial Sloan-Kettering Cancer Center
Education: MD, Catholic University Medical School, 1997; PhD, Open University in London and the European Institute of Oncology, 2003
Recommended by: Thomas Kelly and Harold Varmus

Andrea Ventura has come a long way since he wanted to be an astronomer, but as a newly established scientist getting his own lab off the ground his curiosity into the workings of the world around him is just as much a motivator as it was then.

Ventura originally set out to become a medical doctor, but after receiving his MD in Rome, he shifted gears toward research, eventually completing his PhD at the European Institute of Oncology and the Open University in London. After that, he headed to MIT, where he spent almost six years doing postdoctoral work with Tyler Jacks in the school's Center for Cancer Research. "That's where I learned a lot of molecular biology," Ventura says.

In the Jacks lab, Ventura focused on microRNAs — specifically on a subset of them "for which we have some evidence that they play a role in suppressing cancer," he says. Since that time, he has focused heavily on this cluster of miRNAs known as oncomir-1.

After finishing his postdoc in 2008, Ventura was intrigued by the Memorial Sloan-Kettering Cancer Center, which had just built a new research facility called the Sloan Kettering Institute. "Clearly they are supporting young investigators a lot," he says — and that's where he wound up joining the faculty and setting up his own laboratory.

In that lab, Ventura continues to follow his miRNA research path. His team is creating mouse models and removing individual miRNAs from their genomes to find out what each RNA snippet is actually doing. "At the same time we make mice that express higher levels of microRNAs" to home in on the tumor suppressor and promoter function even more, he adds. The ultimate goal of all of this, he notes, is to "see whether we can create novel anticancer therapies. … Can we induce tumor regression? Our preliminary results are very promising."

Looking ahead

Going forward, Ventura plans to expand his lab personnel, as well as continue his research into this family of miRNAs, and, particularly, into whether there are pharmacological approaches to targeting those mi-RNAs. He's also looking to expand the scope of his investigations to "other microRNAs that we suspect are involved in tumorigenesis … and others that act as tumor suppressors," he says. Since a single miRNA can influence the expression of hundreds of different genes, he adds, it will be critical to establish which of these small RNAs "are functionally important in suppressing or promoting cancer."

Publications of note

In the 2008 paper, "Targeted deletion reveals essential and overlapping functions of the miR-17~92 family of miRNA clusters," published in Cell, Ventura and his co-authors delve into highly conserved miRNAs that have been linked to human cancer. In the study, they knocked out a particular miRNA in mice and found that the mice died "shortly after birth with lung hypoplasia and a ventricular septal defect," they write in the abstract. The group concludes that the overall results "provide key insights into the physiologic functions of this family of microRNAs and suggest a link between the oncogenic properties of miR-17 ... and its functions during B lymphopoiesis and lung development."

And the Nobel goes to...

One of the things Ventura finds compelling about science is that "you never know" where an experiment might take you. He cites the discovery of microRNAs as a good example of an entire field founded on a surprising turn of events and says that in the long term, he would like to figure out exactly how miRNAs function. As for a precise reason to win the Nobel, Ventura says, the element of scientific surprise appeals to him too much to guess.

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