Two scientists won the Nobel Prize in Chemistry Wednesday for developing a way of editing genes likened to “molecular scissors” that offer the promise of one day curing inherited diseases.
Working on opposite sides of the Atlantic, Frenchwoman Emmanuelle Charpentier and American Jennifer A. Doudna came up with a method known as 'CRISPR-cas9' that can be used to change the DNA of animals, plants and microorganisms with extremely high precision. It is the first time two women have won the chemistry Nobel together.
Their work allows for laser-sharp snips in the long strings of DNA that make up the “code of life,” allowing scientists to precisely edit specific genes to remove errors which lead to disease.
“There is enormous power in this genetic tool, which affects us all,” said Claes Gustafsson, chair of the Nobel Committee for Chemistry. "It has not only revolutionised basic science, but also resulted in innovative crops and will lead to groundbreaking new medical treatments.”
Gustafsson said that, as a result, any genome can now be edited “to fix genetic damage.”
But he cautioned that the "enormous power of this technology means we have to use it with great care."
It has already raised serious ethical questions. Most of the world became more aware of CRISPR in 2018, when Chinese scientist He Jiankui revealed he had helped make the world’s first gene-edited babies, to try to engineer resistance to future infection with the AIDS virus. His work was denounced worldwide as unsafe human experimentation because of the risk of causing unintended changes that can pass to future generations, and he’s currently in prison.
In September, an international panel of experts issued a report saying it’s still too soon to try to make genetically edited babies because the science isn’t advanced enough to ensure safety, but they mapped a pathway for any countries that want to consider it.
Charpentier, 51, spoke of the shock of winning.
“Strangely enough I was told a number of times [that I’d win], but when it happens you’re very surprised and you feel that it’s not real,” she told reporters by phone from Berlin after hearing of the award, announced in Stockholm by the Royal Swedish Academy of Sciences. "But obviously it’s real, so I have to get used to it now.”
“I was very emotional,” she added.
When asked about the significance of two women winning, Charpentier said that while she considers herself first and foremost a scientist, she hoped it would encourage others.
“I wish that this will provide a positive message to young girls who would like to follow the path of science," said Charpentier, who is currently the director of the Max Planck Unit for the Science of Pathogens in Berlin.
Doudna told The Associated Press of her own surprise — including that she learned she'd won from a reporter.
“I literally just found out, I’m in shock," she said. "I was sound asleep.”
“My greatest hope is that it’s used for good, to uncover new mysteries in biology and to benefit humankind,” said Doudna, who is affiliated with the University of California, Berkeley and is paid by the Howard Hughes Medical Institute, which also supports AP’s Health and Science Department.
The breakthrough research done by Charpentier and Doudna was only published in 2012, making the discovery very recent compared to many Nobel wins that are often only honoured after decades have passed.
Pernilla Wittung-Stafshede, a member of the Nobel Committee, noted that the method developed by the two biochemists has revolutionised the life sciences.
“The genetic scissors were discovered just eight years ago, but have already benefited humankind greatly,” she said.
The Broad Institute at Harvard and MIT have been in a long court fight over patents on CRISPR technology, and many other scientists did important work on it, but Doudna and Charpentier have been most consistently honoured with prizes for turning it into an easily usable tool.
Dr. Francis Collins, who led the drive to map the human genome, said CRISPR “has changed everything” about how to approach solutions to diseases with a genetic cause, such as sickle cell disease.
“You can draw a direct line from the success of the human genome project to the power of CRISPR-cas to make changes in the instruction book,” said Collins, director of the National Institutes of Health that helped fund Doudna’s work.
The prestigious award comes with a gold medal and prize money of 10 million kronor (more than US$1.1 million), courtesy of a bequest left more than a century ago by the prize’s creator, Swedish inventor Alfred Nobel. The amount was increased recently to adjust for inflation.
On Monday, the Nobel Committee awarded the prize for physiology and medicine to Americans Harvey J. Alter and Charles M. Rice and British-born scientist Michael Houghton for discovering the liver-ravaging hepatitis C virus. Tuesday's prize for physics went to Roger Penrose of Britain, Reinhard Genzel of Germany, and Andrea Ghez of the United States for their breakthroughs in understanding the mysteries of cosmic black holes.
The other prizes are for outstanding work in the fields of literature, peace and economics.
by DAVID KEYTON, CHRISTINA LARSON & FRANK JORDANS, Associated Press