A life-changing discovery: CRISPR scientists awarded the 2020 Nobel Prize in Chemistry

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After several years of nominations, Emmanuelle Charpentier and Jennifer Doudna have finally been awarded the 2020 Nobel Prize in Chemistry for their development of CRISPR/Cas genetic editing. These ‘genetic scissors’ can be used to can change the DNA of animals, plants and microorganisms with unprecedented precision. CRISPR technology has already had a revolutionary impact on the life sciences, but the technique and the Nobel prize itself aren’t without controversy.

By Amy LeBlanc

It is humbling to think that for many of humanities greatest technical innovations, nature usually got there first. This is certainly the case for the gene editing tool CRISPR/Cas, which is based on the viral defense system of bacteria and archaea. Although many scientists were part of the discovery and development process for CRISPR/Cas, it is the seminal work of Emmanuelle Charpentier (Max Planck Unit for the Science of Pathogens, Institute for Infection Biology) and Jennifer Doudna (University of California, Berkeley) that has been recognized in the 2020 Nobel Prize for Chemistry. In the eight years since the publication of their revolutionary 2012 Science paper, the impact of Charpentier and Doudna’s discovery has transformed the life sciences, making genome editing commonplace in laboratories around the world. The breakthrough is already being hailed as one of the key life sciences discoveries of the 21st century.

What is CRISPR/Cas?

As so often the case in basic science, the discovery of CRISPR/Cas was serendipitous. French scientist Emmanuelle Charpentier had been studying Streptococcus bacteria when she discovered a previously unknown molecule, tracrRNA. Her research found that tracrRNA is a part of the bacteria’s immune system, CRISPR/Cas, that disarms viruses by cutting their DNA.

The ability to precisely alter DNA has provided humanity with a tool to easily rewrite the code of life.

After publishing her findings in 2011, Charpentier reached out to Jennifer Doudna, an American biochemist with vast knowledge of RNA. The pair met at a cafe in Puerto Rico while attending a scientific conference, and immediately struck up a collaboration. Together, they succeeded in recreating CRISPR/Cas in a test tube and simplifying the genetic scissors’ molecular components so that they were easier to use. In an era-changing experiment, they then reprogrammed the genetic scissors to not only recognize DNA from viruses, but to also be able to cut any DNA molecule at a controlled, predetermined site. Using this method, DNA can be tailored to delete existing genes or even add new genes to a living cell.

Endless applications

The ability to precisely alter DNA has provided humanity with a tool to easily rewrite the code of life. While genome editing has been previously possible using various methods, older techniques were simply too inaccurate, inefficient or impractical for large scale implementation. Since Charpentier and Doudna published their work on the CRISPR/Cas tool in 2012, the use of these genetic scissors has exploded.

The potential of CRISPR/Cas is only limited by human ingenuity. However, with such a powerful gene editing tool, unethical applications have also become possible.

The possibilities seem endless, with applications already underway in most branches of the life sciences. Scientists conducting basic research are using the method to improve their research models. Plant researchers are applying the tool in agriculture, to develop crops that can withstand disease and climate change effects like drought. In medicine, CRISPR/Cas has been used to develop a whole new line of cancer treatments, with many clinical trials underway. And, perhaps most astounding, for the first time in human history we have the tools at our disposal to potentially cure inherited diseases like blood disorders and hereditary blindness.

Read this previous BioVox article to learn about Inari: a company using gene editing to create more resilient crops.

In many ways, the potential of CRISPR/Cas is only limited by human ingenuity. However, with such a powerful gene editing tool, unethical applications have also become possible. In 2018, the world was shocked Chinese researcher He Jiankui revealed that he had used CRISPR to create the world’s first gene-edited babies, in an attempt to make the children resistant to HIV. The announcement was immediately followed by widespread condemnation from the global scientific community, including from Doudna herself, who called the work horrifying, risky and premature. He Jiankui is now serving a prison sentence for illegal medical practices, and Doudna continues to be active in encouraging the international scientific community to develop sound guidelines for the use of CRISPR. Like any powerful tool, CRISPR/Cas has the potential to cause damage if used maliciously, rashly or carelessly. It is up to everyone to see that regulations are put in place for the ethical and beneficial use of this incredible technique.

A controversial prize

Though nobody is arguing the fact that Charpentier and Doudna were well-deserving of the Nobel Prize, it is important to recognize that this discovery was not made in isolation. Though nominations have been made for CRISPR to be recognized by the Nobel Prize committee for half a decade, multiple names have been put forth for the honor. Indeed, several of these scientists have been recognized in previous awards, including Spanish microbiologist Francisco Mojica, who together with Dutchman Ruud Jansen named CRISPR in 2000 (CRISPR stands for ‘clustered regularly interspaced short palindromic repeats’). Biochemist Virginijus Šikšnys from Lithuania has also previously shared a prize with Charpentier and Doudna, as has Chinese-American geneticist Feng Zhang who, in an early 2013 Science paper, modified the CRISPR/Cas system to make precise genome cuts in human and mouse cells. And that’s not to mention the countless others, researchers, graduate students and technicians, who contributed to this body of work over the years.

The recognition of Charpentier and Doudna’s work on CRISPR/Cas is an important win for the global scientific community, both as an acknowledgement of the importance of this revolutionary advancement in life sciences, and as a way of championing women scientists.

Precisely because research is so often a group effort, awards like the Nobel Prize have increasingly been criticized for exclusion and glorification of a select few individuals instead of due recognition of all contributors. Discoveries are never made in isolation: the idea of the ‘lone genius’ scientist is not only false but also detrimental to society’s understanding of the scientific process. Added to this is the Nobel Prize’s notoriously poor track record with awardee diversity: in its 119-year history, men have dominated the list of Nobel laureates, with only 3% of the science awardees being women. Ethnic diversity is also severely lacking: though there have been a few laureates of something other than European descent, the ranks are mostly homogenously white. To this day, there has never been a black scientist laureate.

Read this previous BioVox article to learn more about the ethics and potentials of CRISPR applications.

We need to bear in mind the traps and limitations of awards like the Nobel Prize. Yet, there is still room for prizes such as these in promoting the importance and societal value of scientific discoveries. And with greater diversity in laureates, we provide the next generation with role models that more accurately reflect what a scientist might look like. With that in mind, the recognition of Charpentier and Doudna’s work on CRISPR/Cas is an important win for the global scientific community, both as an acknowledgement of the importance of this revolutionary advancement in life sciences, and as a way of championing women scientists.

Header image © Johan Jarnestad/The Royal Swedish Academy of Sciences