MicroRNA Pioneers Win Medicine Nobel

"The Nobel Prize in medicine was awarded to Victor Ambros and Gary Ruvkun for the discovery of microRNA, molecules that help control how genes are expressed.

Their findings unlocked new areas of research into the roles these molecules play in human health. Researchers are exploring microRNA treatments for cancer, heart disease and dementia.

Ambros and Ruvkun were postdoctoral fellows in the 1980s in the laboratory of biologist Robert Horvitz, who won the Nobel Prize in 2002 for his research in gene regulation. In Horvitz's lab, they studied the roundworm C. elegans to better understand the role genes play in the development of different cell types.

Researchers already knew genes are copied to make molecules called mRNA that are translated into proteins that help determine cell type. 

The discovery of microRNA, which are about a hundredth the size of typical mRNA, added new understanding to how genes can be turned off.

Ambros, with the help of research assistant Rosalind Lee -- who also happens to be his wife -- and geneticist Rhonda Feinbaum, discovered the first microRNA in 1993. It appeared to be responsible for a mutation in the worms he studied in the lab.

Ambros wondered whether the finding was limited to worms. But a few years later Ruvkun, who was a close friend of Ambros, found another example of microRNA, one in humans and many other creatures.

"I had to reorganize my view of the world," Ambros, now 70 years old, said.

Researchers have since found microRNA in animals, plants and some viruses. Hundreds have been found in people. The molecules can bind to mRNA and destabilize it. They can also inhibit the amount of protein made from mRNA.

Lab experiments have shown that when microRNAs go awry, blindness, deafness, cancer and death can occur, said David Bartel, a biologist at the Massachusetts Institute of Technology.

"These are really important in mammalian development, physiology and disease," said Bartel. He co-founded Alnylam Pharmaceuticals to develop drugs using microRNA-like molecules to treat rare genetic disorders and high cholesterol.

Other companies are exploring whether blocking certain microRNAs could treat diseases.

Ruvkun and Ambros were giddy with excitement after learning of their Nobel honors.

Ruvkun, 72 and a genetics professor at Harvard Medical School, said he was fast asleep when the Nobel committee called in the early hours. "There have been other awards," he said. "The amount of attention a Nobel attracts is a hundredfold more."

Ambros was also asleep when the Nobel committee tried and failed to reach him. His son ended up breaking the news. "He said, 'If you get a call from somebody in Sweden, answer it!' " said Ambros, who is at UMass Chan Medical School.

Ruvkun and Ambros have been good friends since first meeting in Horvitz's lab. Ambros used to fly small planes and took Ruvkun on many adventures. They said they look forward to celebrating their win together soon.

"I don't have words for what it means to me. It's just unreal," Ambros said, with his wife -- who goes by Candy -- by his side. The couple met as undergraduates at MIT and have been colleagues for almost 40 years.

"We didn't go into science for fame or fortune. It was just curiosity," Lee said." [1]

1.  U.S. News: MicroRNA Pioneers Win Medicine Nobel. Mosbergen, Dominique.  Wall Street Journal, Eastern edition; New York, N.Y.. 08 Oct 2024: A.3.