Breakthrough Prizes Highlight CRISPR Gene Editing, Gene Regulation

By Bio-IT World Staff

November 10, 2014 | Emmanuelle Charpentier and Jennifer Doudna, who together showed in a celebrated 2012 paper in Science that the bacterial CRISPR-Cas9 system for fighting viruses could be used as a simple and flexible tool for genetic engineering, are among the winners of the 2015 Breakthrough Prizes in the Life Sciences. In the two years since Charpentier and Doudna's basic discovery, CRISPR gene editing has become one of the fastest-growing biological tools of all time, allowing non-specialist labs to conduct gene engineering experiments and spawning countless suggested applications. Both the discoverers have become involved in startups seeking to use CRISPR to cure genetic diseases in humans: Charpentier as a scientific founder of CRISPR Therapeutics, and Doudna as a founder of Editas Medicine. (See, "Gene Therapy's Next Generation.") It has also been suggested that CRISPR could become a powerful solution to the antibiotic resistance crisis, literally carving antibiotic resistance genes out of bacterial genomes.

The Breakthrough Prizes, although a relatively new fixture in honoring scientific achievements, are often compared to the Nobels for their emphasis on foundational discoveries and the life-changing size of the awards. The Breakthrough Prizes, however, are more likely to single out recent accomplishments of great promise but untested value, where the Nobels tend to recognize years- or decades-old work that has gradually proved fundamental to whole fields of research. This more forward-looking approach may partly be made possible by the much deeper purse of the Breakthrough Prizes, which in the Life Sciences category alone award $3 million each to up to six honorees a year. By comparison, the Nobels recognize just one discovery in each category, encouraging the Nobel committee to select work of established value. Nobel laureates, up to three per category, share a purse that was worth roughly $1.2 million in 2014.

The Breakthrough Prizes, founded in 2012 by Russian tech investor Yuri Milner, originally recognized groundbreaking research in physics, but other tech billionaires quickly signed on to extend the awards to the life sciences and mathematics. Today, eight founders contribute to the Life Sciences prizes: Milner and his wife Julia; Facebook founder Mark Zuckerberg and his wife Priscilla Chan; Alibaba founder Jack Ma and his wife Cathy Zhang; and Sergey Brin and Anne Wojcicki, respectively a Google co-founder and the CEO of direct-to-consumer genetic testing company 23andMe. The founders have designed the awards to have a particular emphasis on fighting rare diseases and diseases of aging, and one prize a year is set aside for research relevant to the treatment of Parkinson's disease.

The prizes were given out at an opulent event held on Sunday night in Mountain View, California, uniting tech moguls, Hollywood celebrities and scientific icons as part of the Breakthrough Prizes' mission to increase the cultural caché of scientific research. The celebrity gala atmosphere of the ceremonies, along with the jaw-dropping size of the awards, has opened the Prizes up to the criticism of being frivolous or lavishing money on individuals that could be better invested in research projects. Previous winners, however, have invested portions of their prizes in initiatives like the Tri-Institutional Breakout Awards for postdoctoral researchers. Each winner is also obligated to serve as a judge for the next year's prizes, helping to ensure that awards go to the achievements that are most exciting to working scientists. The current Chair of the Breakthrough Prize in the Life Sciences Board, Cornelia Bargmann, received her prize in 2013 for exploring the molecular pathways involved in forming synpatic connections between neurons during brain development.

Whatever one's personal feelings about the Breakthrough Prizes, few would begrudge the awards to Charpentier and Doudna, whose work with CRISPR has made it far easier than ever before to edit the genomes of living organisms, over a remarkably short period of time. The use of genetic material to control cellular activity was a major theme of this year's prizes, with additional awards given to Victor Ambros and Gary Ruvkun for the discovery of microRNA and its role in regulating gene expression, and C. David Allis for his work with epigenetic regulatory factors. The sixth honoree, Alim-Louis Benabid, is a neurosurgeon who discovered that electrical stimulation of the brain could help control tremors and other symptoms in Parkinson's patients, after incidentally observing the effects during surgical procedures.