Three Boston-area researchers win prestigious Breakthrough awards


Three Boston-area researchers received prestigious Breakthrough Prizes for their groundbreaking work in gene editing, diabetes treatment, and multiple sclerosis research.
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One of the prizes, awarded to Liu, who conducts his research at The Broad Institute in Cambridge, recognizes his lab’s discovery of base and prime gene editing to fight genetic diseases. The technology corrects DNA mutations without the need for cutting DNA’s double-helix structure like previous technologies have. Base editing, developed by Liu’s lab in 2016, corrects single-letter “misspellings” in DNA, and his lab’s 2019 invention of prime editing replaces full stretches of faulty DNA with corrected versions.

Liu’s technology has been distributed to more than 20,000 non-profit labs worldwide that are working on treatments for a wide range of genetic diseases, many of which are rare diseases that often do not get attention from large pharmaceuticals. The technology makes it “economically feasible to create therapeutics for small markets,” according to a news release.

Fifteen clinical trials of the technology are ongoing in five different countries, and life-saving results have been seen in patients with T-cell leukemia, sickle-cell disease, beta-thalassemia, and high cholesterol have already been seen.

“It was really a huge team effort,” said Liu. “It’s a wonderful testament to the the hard work of many of the the people who I’ve had the honor of working with over the past 26 years.”

Liu intends to donate his prize winnings to fund research in other labs that might be struggling from federal funding cuts.

Habener along with four fellow researchers will share in another of the prizes for the discovery of GLP-1 medications in treating diabetes and obesity. The drugs mimic the GLP-1 hormone in the body, which regulates blood sugar and appetite.

“I never thought my dream would come true to discover something that impacts millions, even billions, of lives,” said Habener, who researched at Harvard University and Massachusetts General Hospital.

The award is being shared by Daniel J. Drucker, Jens Juul Holst, Lotte Bjerre Knudsen, and Svetlana Mojsov. Drucker, who is now at the University of Toronto, and Mojsov, at Rockefeller University, both contributed to the discovery while they were at Harvard and MGH.

The team’s invention, which spanned over decades of research, established a robust market for the medications.

A third prize will go to Harvard researcher Ascherio and the University of California’s Stephen L. Hauser, for their work finding the virus connected to risk for multiple sclerosis. Those findings may lead to the possibility to treat the disease earlier with antiviral drugs, making it more manageable, or the development of a vaccine to stop the disease at its root.

Ascherio is now looking to how the discovery could be built upon to start fighting other diseases before they take hold.

“We are desperately trying to get funding, to expand our research to apply the same blueprint of study design to investigate the role of infection in other diseases, which is a major question that is unanswered at this point,” said Ascherio.

Ascherio found all people who develop multiple sclerosis must have had Epstein Barr Virus previously, which is one of the most common human viruses and the cause of infectious mononucleosis, also known as “mono.” Many people contract the disease, especially in childhood, without ever knowing they had it, and contracting the disease multiplies the risk for developing MS by 32 times.

Maren Halpin can be reached at maren.halpin@globe.com.

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