Gene editing tool CRISPR wins the chemistry Nobel

Turning a bacterial defense mechanism into one of the most powerful tools in genetics has earned Jennifer Doudna and Emmanuelle Charpentier the Nobel Prize in chemistry. 

The award for these genetic scissors, called CRISPR/Cas 9, is “a fantastic prize,” Pernilla Wittung-Stafshede, a member of the Nobel Committee for Chemistry, said at an Oct. 7 news conference held in Stockholm by the Royal Swedish Academy of Sciences to announce the prize. “The ability to cut the DNA where you want has revolutionized the life sciences. We can now easily edit genomes as desired — something that before was hard, or even impossible.”  

“The genetic scissors were discovered just eight years ago, but have already benefited humankind greatly,” she said. “Only imagination sets the limits for what this chemical tool … can be used for in the future. Perhaps the dream of curing genetic diseases will come true.” She later amended the statement to say that ethics and law are also important to determine what can and should be done with the tool, as some human gene editing is extremely controversial.

Only five other women have ever won the Nobel Prize in chemistry. “I wish that this would provide a positive message specifically to the young … girls who would like to follow the path of science, and I think to show them that women in science can also be awarded prizes, but more importantly that women in science can also have an impact through the research that they are performing,” Charpentier said in response to a question during the news conference.

The two will split prize money of 10 million Swedish kronor, about $1.1 million.

Emmanuelle Charpentier (left) and Jennifer Doudna (right) teamed up to turn a bacterial defense system into a gene editor.From left: ©Helmholtz/Hallbauer&Fioretti; Sam Willard/Sam Willard Photography, Berkeley

The tool, a programmable molecular scissors known as CRISPR/Cas9, has been used by bacteria and archaea for millions to billions of years to fight viruses (SN: 4/5/17).

CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats. In essence, these short, repeating bits of DNA sandwich bacteria’s version of the FBI’s most wanted list — invading viruses. Every time bacteria encounter a virus, they take a DNA mugshot of it and file it in between the repeats. The next time the bacteria encounters that virus, they make RNA copies of the mugshots. Those RNA photocopies then team up with another bit of RNA known as a trans-activating CRISPR RNA, or tracrRNA, to form an all-points bulletin known as a guide RNA. Guide RNAs shepherd the DNA-cutting enzyme Cas9 to the virus, where the enzyme chops and eliminates the threat.

Doudna of the University of California, Berkeley, and Charpentier, now director of the Max Planck Institute for Infection Biology in Berlin, turned CRISPR/Cas9 from a bacterial defense system into a gene editor. Their innovation was to fuse the mug shot RNA to the tracrRNA, creating a single guide RNA. And the researchers realized that the mugshots didn’t have to be molecular pictures of viruses. Instead, by replacing the mugshot with RNA that matches a gene, the scientists could direct Cas9 to snip that gene — or any gene, really.

These researchers and other scientists have taken these genetic scissors to the next step, using CRISPR/Cas9 to cut and edit genes in human cells. Scientists rave about how cheap, versatile and easy to use CRISPR is. Researchers have used it edit genes in a wide variety of animals, including dogs (SN: 8/30/18), mice (SN: 1/26/17), butterflies (SN: 8/24/16), cows (SN: 2/3/17), pigs (SN: 8/10/17), snails (SN: 5/14/19) and mosquitoes. 

The tool has also been used to encode data and store movies in bacterial DNA (SN: 7/12/17). Plants and mushrooms have gotten the CRISPR treatment, too. And the gene editor has been used to reprogram human immune cells to fight cancer (SN: 11/16/16) and to turn cancer cells against each other (SN: 7/11/18).

With CRISPR’s great power comes great controversy, Doudna warned in her 2017 book A Crack in Creation with coauthor Samuel Sternberg. While the gene editor might be used to stamp out invasive species and prevent mosquitoes from carrying disease, it might also drive entire species extinct or create ecological disasters. Already scientists have wiped out small populations of mosquitoes in the laboratory using a CRISPR-based molecular copy machine known as a gene drive (SN: 9/24/18).

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Most controversially, a scientist in China edited genes in human embryos, producing twin baby girls in 2018 (SN: 11/28/18). Backlash against his actions was swift and vocal. But many people fear the door is already open to “designer babies,” health care inequalities and other abuses (SN: 12/17/18).

“This enormous power of this technology means that we need to use it with great care,” Claes Gustafsson, chair of the Nobel Committee for Chemistry, said at the news conference. “But it’s equally clear that this is a technology… that will provide humankind with great opportunities.” 

More hopefully, clinical trials testing CRISPR/Cas9’s ability to treat cancer, sickle cell disease, beta-thalassemia and inherited blindness began in 2019 (SN: 8/14/19). If successful, CRISPR/Cas9 may provide therapies, or even cures, for previously untreatable genetic conditions.

CRISPR has also played a role in the coronavirus pandemic, with CRISPR-based diagnostic tests for COVID-19 (SN: 8/31/20) and therapies in development.  

Nearly all scientific prizes for CRISPR/Cas9 have honored Doudna and Charpentier. Some prizes have also included Feng Zhang of the Broad Institute of MIT and Harvard, who holds the patent on using the gene editor to make changes in eukaryotic cells, including human  and animal cells. Many people thought that the prize would not honor work on CRISPR until the patent dispute was settled. (Zhang is a member of the board of trustees for the Society for Science & the Public, an educational nonprofit in Washington, D.C., that also publishes Science News. He is also an alumnus of the Society’s Regeneron Science Talent Search.) 

Two other scientists, Rodolphe Barrangou of North Carolina State University in Raleigh and Philippe Horvath of DuPont Nutrition & Biosciences in Dangé-Saint-Romain, France, have also been honored for discoveries related to CRISPR. The duo discovered CRISPR’s natural role as a bacterial immune system while working with yogurt bacteria at the food ingredient company Danisco.

And two major prizes — the Warren Alpert Foundation prize and the Kavli prize for neuroscience — have honored Virginijus Šikšnys, a biochemist at Vilnius University in Lithuania. Šikšnys authored an independent paper describing the same innovation made by Doudna and Charpentier that was held up in the publishing process, and didn’t hit presses until three months after the UC Berkeley team’s report.

When asked if other scientists had been considered for the prize, Gustaffson said, “this is a question we never answer. We are just extremely happy for this year’s laureates. It’s a big field, and there’s a lot of good science being done.”

 Staff writer Maria Temming contributed to this story.