Doudna and Charpentier win Chemistry Nobel for CRISPR/Cas9 method of gene editing

This year’s Nobel Prize in Chemistry was long anticipated, for the CRISPR/Cas9 system of gene editing was a tremendous accomplishment in biology and chemistry. It promises a lot, including curing human genetic disease (see the first five posts here). Remember, Nobel Prizes in science are designed to reward those who made discoveries potentially helping humanity, not those who just made general scientific advances.

A prize for developing the editing system was, then, almost inevitable. The only question was “who would get it?”, since several people contributed to the work that led to CRISPR/Cas9.  It turns out that the Prize—in Chemistry—went to the two frontrunners, Jennifer Doudna of UC Berkeley and Emmanuelle Charpentier at the Max Planck Institute for Infection Biology in Berlin.  Other serious contenders were George Church of Harvard, Virginijus Šikšnys at the Vilnius University of Biotechnology, Francisco Mojica of the University of Alicante, and Feng Zhang of the Broad Institute (the dispute was largely over whether those who developed ways to use the method in human cells also deserved the Prize). There will be a lot of kvetching today, but if I had had to pick two to get the prize, given that only three can get it au maximum, it would be Doudna and Charpentier. (They could have awarded up to six prizes if they’d split the CRISPR award between Physiology or Medicine and Chemistry.)

The press release from the Nobel Foundation says this:

Genetic scissors: a tool for rewriting the code of life

Emmanuelle Charpentier and Jennifer A. Doudna have discovered one of gene technology’s sharpest tools: the CRISPR/Cas9 genetic scissors. Using these, researchers can change the DNA of animals, plants and microorganisms with extremely high precision. This technology has had a revolutionary impact on the life sciences, is contributing to new cancer therapies and may make the dream of curing inherited diseases come true.

Researchers need to modify genes in cells if they are to find out about life’s inner workings. This used to be time-consuming, difficult and sometimes impossible work. Using the CRISPR/Cas9 genetic scissors, it is now possible to change the code of life over the course of a few weeks.

“There is enormous power in this genetic tool, which affects us all. It has not only revolutionised basic science, but also resulted in innovative crops and will lead to ground-breaking new medical treatments,” says Claes Gustafsson, chair of the Nobel Committee for Chemistry.

As so often in science, the discovery of these genetic scissors was unexpected. During Emmanuelle Charpentier’s studies of Streptococcus pyogenes, one of the bacteria that cause the most harm to humanity, she discovered a previously unknown molecule, tracrRNA. Her work showed that tracrRNA is part of bacteria’s ancient immune system, CRISPR/Cas, that disarms viruses by cleaving their DNA.

Charpentier published her discovery in 2011. The same year, she initiated a collaboration with Jennifer Doudna, an experienced biochemist with vast knowledge of RNA. Together, they succeeded in recreating the bacteria’s genetic scissors in a test tube and simplifying the scissors’ molecular components so they were easier to use.

In an epoch-making experiment, they then reprogrammed the genetic scissors. In their natural form, the scissors recognise DNA from viruses, but Charpentier and Doudna proved that they could be controlled so that they can cut any DNA molecule at a predetermined site. Where the DNA is cut it is then easy to rewrite the code of life.

Since Charpentier and Doudna discovered the CRISPR/Cas9 genetic scissors in 2012 their use has exploded. This tool has contributed to many important discoveries in basic research, and plant researchers have been able to develop crops that withstand mould, pests and drought. In medicine, clinical trials of new cancer therapies are underway, and the dream of being able to cure inherited diseases is about to come true. These genetic scissors have taken the life sciences into a new epoch and, in many ways, are bringing the greatest benefit to humankind.

I haven’t looked it up, but I think this is the first time that two women have been the sole recipients of any Nobel prize.(Correction: I should have said “Prize for Science”, for, as a reader pointed out below, two women shared the 1976 Nobel Peace Prize: Betty Williams and Mairead Corrigan. Their achievement was organizing to suppress sectarian violence during the Troubles in Northern Ireland.

Here are Doudna and Charpentier from the Washington Post (the paper’s caption):

Here’s the live stream of the announcement from Stockholm. The action begins at 11:45 with the announcement in English and Swedish, and the scientific explanation starts at 19:10.

Once again, although seven people, including Matthew, guessed the winners in our Nobel Prize contest (here and here), nobody got the Chemistry or Physics prizes. Given your miserable failures, I may have to have contest for the literature prize alone.

Matthew was also prescient in his book, Life’s Greatest Secret (2015), which includes this sentence:

“Whatever happens next, I bet that Doudna and Charpentier—and maybe Zhang and Church—will get that phone call from Stockholm.”

In 2017, I reviewed (favorably) Jennifer Doudna’s new book on CRISPR, A Crack in Creation, for the Washington Post. (Samuel Sternberg was the book’s co-author). The book is well worth reading, but I did have one beef connected not with the narrative, but with where the dosh goes for this discovery. Here’s what I wrote then:

. . . this brings us to an issue conspicuously missing from the book. Much of the research on CRISPR, including Doudna’s and Zhang’s, was funded by the federal government — by American taxpayers. Yet both scientists have started biotechnology companies that have the potential to make them and their universities fabulously wealthy from licensing CRISPR for use in medicine and beyond. So if we value ethics, transparency and the democratization of CRISPR technology, as do Doudna and Sternberg, let us also consider the ethics of scientists enriching themselves on the taxpayer’s dime. The fight over patents and credit impedes the free exchange among scientists that promotes progress, and companies created from taxpayer-funded research make us pay twice to use their products.

. . . . Finally, let us remember that it was not so long ago that university scientists refused to enrich themselves in this way, freely giving discoveries such as X-rays, the polio vaccine and the Internet to the public. The satisfaction of scientific curiosity should be its primary reward.

I’m not sure how the legal battle between the participants (via Berkeley and MIT) has shaken out, and can’t be arsed to look it up, but surely a reader or two will know