Emmanuelle Charpentier and Jennifer A. Doudna will receive the 2020 Nobel Prize in Chemistry for discovering one of gene technology’s sharpest tools: the CRISPR/Cas9 genetic scissors. Professor Emmanuelle Charpentier, 2016 L’Oréal-UNESCO For Women in Science Laureate for Europe and Professor Jennifer Doudna, 2016 L’Oréal-UNESCO For Women in Science Laureate for North America, each contributed key insights to the development of this ground breaking new technology that has set the scientific world on fire, reinventing genetic research and making it possible to perform microsurgery on DNA, the genetic material of plants, animals and humans. Working in collaboration, the two researchers discovered an easy way to alter any organism’s DNA. Known as CRISPR-Cas9, this genome editing technique enables scientists to remove and add pieces of genetic material with exquisite precision. It can be used to disable genes, correct genetic disorders or to insert genes to create animal models of human disease.
We are only just beginning to grasp the full impact of this extraordinary new technology. The CRISPR-Cas9 complex is able to home in on a matching sequence with extraordinary precision. It opens completely new possibilities in gene therapy, cell therapy and immunotherapy. It opens new fields in agriculture and biotechnology. It offers new means of developing medicines. It offers the possibility of removing faulty disease-causing DNA, for instance in cells in the lungs of children affected with cystic fibrosis or the muscles of those with some forms of muscular dystrophy. It has already been used to save the life of a child with an incurable form of leukaemia and to improve the sight of patients suffering from retinitis Pigmentosa, a genetic eye disease. And there is much, much more to come.
Modern science, defined by collaboration
This important breakthrough provides the ideal example of what scientists achieve when working together.
Professor Charpentier is a highly respected microbiologist and geneticist. For some time she had been working on Streptococcus pyogenes, more familiar to us as one of the bacteria responsible for sore throats. In particular she was fascinated by the way that it defended itself against attack by phages, viruses that hunt down bacteria. Professor Doudna is a world renowned structural biologist and understanding RNA, a close cousin of DNA, has long been her forte. Back in 2005 she was asked to have a look at intriguing repeating regions of DNA in bacteria called CRISPR sequences.
For today’s scientists, crossing borders between countries and disciplines is instrumental to opening doors to new questions and new answers.
Professor Emmanuelle Charpentier
In 2011, Emmanuelle Charpentier asked Jennifer A. Doudna to collaborate. Professor Charpentier had already published important work showing that bacteria were effectively able to vaccinate themselves against types of virus that they had already encountered using a CRISPRCas system. Together, Doudna, with her profound knowledge of RNA and Charpentier, with her CRISPR-Cas insight, published findings demonstrating that the mechanism used by bacteria to disable their foes could be adapted as a programmable precision genetic tool to modify genes in cells and organisms.
Collaborations have been a crucial part of the success of my scientific research throughout my career. Multidisciplinary research teams are essential to making fundamental progress in any field. Indeed, collaboration defines the modern mode of scientific research.
Professor Jennifer A. Doudna
In 2016, they both received a L’Oréal-UNESCO For Women in Science International Award, which is presented every year to five outstanding women scientists in recognition of their scientific accomplishments.
Professor Charpentier has been immensely generous with her time, helping other scientists understand how to use CRISPR-Cas9 technology in their work. As a result, its use has spread like wildfire in the science community. It has literally reinvented genetic research.
Both scientists quickly realized that gene editing raised many potential ethical concerns. Jennifer A. Doudna has been at the forefront of ensuring that these concerns are properly debated and understood. In particular she has pointed out potential concerns posed by so called ‘germ line editing’, that is affecting future generations by altering sequences in either sperm or egg.
Professor Emmanuelle Charpentier
Professor Jennifer A. Doudna
CRISPR – an elegant but leathal weapon
Some types of viruses attack bacteria. They infect them, using them to multiply. But bacteria can fight back. Using molecular scissors, bacteria snip out a piece of DNA from an invading virus. They tuck this ‘souvenir’ of their foe away in the so called CRISPR region of their own DNA which contains many such souvenirs. These are equivalent to stamps on a vaccine record card, effectively recording that the bacteria is now immunized against attack by the viruses it has in its collection. Should one of the viruses appear again, the bacteria quickly uses the right ‘souvenir’ as a template to generate copies made of RNA (a chemical cousin of DNA).
These are the foundation of the bacteria’s lethal and highly specific antivirus weapon. The weapon is then activated by the addition of a further, and essential small piece of RNA, together with a subset of Cas enzyme called Cas9. Like a guided missile, this complex searches through the viral genome for an exact match. Only once it locks on the matching sequence in the virus, does the Cas9 act as molecular scissors, cutting clean through the double helix of the virus’s DNA, disabling it for good.
The L’Oréal-UNESCO For Women in Science International Awards are presented every year to five outstanding women scientists in recognition of their scientific accomplishments.
UNESCO and the L’Oréal Corporate Foundation seek to recognise women researchers who, through the scope of their work, have contributed to overcoming today’s global challenges. Today, only 30% of researchers are women. Stereotypes conceal a significant part of scientific history, and perpetuate gender inequality in the access to these fields of study and careers. We cannot afford to deprive ourselves of the talents of half of humanity: women advance science, and science advances the world.