Jennifer Doudna was sound asleep when her phone began buzzing, so she missed the first few calls. When the incessant ringing finally roused her at 3 a.m., it was a reporter who wanted her reaction to the just-awarded Nobel Prize in Chemistry.
“Who won?” Doudna asked.
“It was a little embarrassing,” Doudna said during a press conference held later in the day when the reporter told her she had been given the prestigious award, along with her collaborator Emmanuelle Charpentier from the Max Planck Institute. “I was really deeply asleep.”
Doudna and Charpentier were jointly awarded the 2020 Nobel Prize in Chemistry for their discovery of CRISPR-Cas9, a genome editing technology that allows scientists to deftly edit DNA from virtually any living thing with a precision and ease never before possible. Since they described the technology in 2012, they and other researchers have studied CRISPR for use in possible treatments for human diseases ranging from cancer to HIV as well as in agriculture for ways to make plants drought and pest resistant. Doudna and Charpentier are the first all-female winners of the Chemistry Prize, and Doudna is the first female faculty member at University of California, Berkeley to earn the honor.
The news was such a shock that Doudna thought the reporter on the phone had been joking—until she answered a second call early Wednesday morning. It was from her former post-doctoral fellow Martin Jinek, who conducted many of the experiments that led to the CRISPR discovery. “Martin would never call unless it was real,” she tells TIME. “We had a really great discussion, and he was really, really happy.”
Doudna says she will never forget the moment that she and Jinek looked at the data from experiments they had run and realized they had found a relatively easy way to cleanly edit DNA—and the vast implications that could have for addressing disease as well as agricultural challenges. “I like to call it a democratizing technology, which is widely available and accessible to scientists globally, and not expensive to get hold of,” she says. “It really opened the door to all sorts of research and increasingly applications that would not have been possible otherwise.”
A colleague at University of California, Berkeley first introduced Doudna to the odd ability that bacteria have of neatly snipping out the genes that viruses rudely insert into bacterial genomes. Because viruses can’t copy their own genomes by themselves, they need to co-opt other organisms, including bacteria, to help them. Intrigued, Doudna began investigating the phenomenon, and soon after met Emmanuelle Charpentier, then in the department of molecular biology at Umea University in Sweden, at a scientific conference in Puerto Rico. Walking the streets of old San Juan, the two scientists indulged their professional imaginations and realized they had complementary skill sets that could speed up the process of unpacking this gene editing mechanism.
Within a year, in 2012, Doudna and Charpentier published their groundbreaking paper describing CRISPR-Cas9, a system that gave researchers the molecular “scissors” to precisely cut DNA wherever they wanted.
Scientists are still finding new clever and innovative ways to apply the technology, and dozens of startup companies, including ones founded by Doudna herself, have sprouted up to exploit the potential. The technology has also led to a years-long patent battle between University of California, Berkeley, and Massachusetts Institute of Technology (MIT); MIT’s Feng Zhang was the first to describe CRISPR in so-called eukaryotic cells, which includes those in plants, animals and people, but published his findings several months after Doudna and Charpentier, whose patent claims cover the technology more broadly. Eric Lander, founding director of the Broad Institute of MIT and Harvard, where Zhang also holds an appointment, tweeted “huge congratulations” to the duo.
The stakes for the patent conflict are high and could mean millions of dollars in licensing revenues for the winner. In eight short years, scientists exploiting CRISPR have figured out how to use it to edit HIV genes out of human cells, cut out a gene responsible for a devastating congenital heart condition, and explore ways to delete cancer-causing genes, all of which could lead to life-saving, and highly sought-after new therapies.
“The field in many ways was ripe for a technology to allow easy manipulation of DNA in precise ways, which is exactly what CRISPR does,” says Doudna. No specialized training or equipment is needed to use the tech; you simply need to license it.
And while the pliability of the technology has indeed had the democratizing effect that Doudna hoped for, it has also made possible abuse and more questionable application. In 2018, for example, Chinese scientist He Jiankui shocked the scientific community when he announced that he had used CRISPR to genetically edit the genomes of human embryos created by a couple in which the father was HIV positive. He altered their genomes to give them genetic protection against infection and then transferred the embryos to the mother for a full-term pregnancy, that led to the live birth of twin girls. The act was widely criticized by leading scientists, including Doudna, and led to recommendations by the U.S. National Academy of Medicine and the UK Royal Society to discourage use of CRISPR in embryos that will be implanted for pregnancy until safety questions can be answered.
Any technology can be abused, says Doudna, but “with CRISPR because it is easily accessible means we have to really be on top of it as a scientific community. There has been a very good international response to that incident that galvanized an international effort to make sure we have a culture of transparency globally, and that as a community we work to ensure responsible use of the technology.”
That may someday even include a more potent way to protect against infectious diseases like the coronavirus that has had such a devastating effect on countries and their economies. The ability to edit genes, including genes in immune cells, using CRISPR, could ultimately lead to the next phase of immune protection beyond vaccines. “I imagine a day when the technology of genome editing could be used to alter immune cells so they are primed to defend against new viruses, and potentially do that very quickly to provide an alternative to having to go through many, many months of developing a new vaccine,” she says. “That’s a future goal—it’s not going to happen next year, but nonetheless it’s something very exciting to think about the future potential of CRISPR.”
In the meantime, Doudna plans to enjoy one of the perks provided by her new honor: a free, personal parking space on the Berkeley campus.±
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Contributor: Alice Park