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Excellence is a choice


The French microbiologist Emmanuelle Charpentier is a pioneer of CRISPR-Cas9 technology, a prize-winning tool for gene editing. Viewed as a revolution in biology, it is already used in a wide variety of applications in laboratories worldwide. Professor Charpentier will present her research at the upcoming EMBO | EMBL Science and Society Conference in November. In an interview with EMBOencounters she speaks about her life-changing discovery and what it is like to suddenly step into the full public spotlight.


How has your life changed after your breakthrough discovery in 2012?

Over the last two years, the research of my laboratory has been awarded with a number of prizes. I am quite amazed by the strong and fast support received from the scientific community for our achievements on CRISPR-Cas9. This all happened while I was establishing my new laboratory in Germany. The change of environment helped me keep my feet on the ground because I needed to deal with substantial activities around my integration in the new institution, such as administrative and logistic issues and recruitments to establish a new team. The prizes surely highlight the fundamental nature of our research that has been translated at dazzling speed into biomedical and biotechnology applications on a large-scale. This is a critical message to convey to the governmental and funding organizations that, once again, it all comes down to basic science: the investigation of new mechanisms, the basis for the development of novel therapeutic strategies or biotechnologies. 


Were you aware at that time that this is a game-changing method?

I understood very quickly that if CRISPR-Cas was to be exploited as a tool for genome silencing and engineering, then the CRISPR-Cas9 system would provide the best opportunities for application – because it is the simplest of the CRISPR-Cas systems existing in bacteria. This was later confirmed by seminal experiments done with my student Krzysztof Chylinski showing that the enzyme Cas9 just needs a duplex of RNA to cleave DNA. I had even predicted early on that the system could be harnessed to treat human genetic disorders, which the Swiss-based company CRISPR Therapeutics that I cofounded together with Rodger Novak and Shaun Foy now focuses on. 


How do you define scientific success?

To me scientific success can be measured on different levels. Surely, ultimate success is linked to an unexpected finding that can result in a scientific breakthrough: to be able to identify a high-impact biological mechanism, not only a purely theoretical one but one that opens up new applications on a global scale. Scientific success can also be measured by activities around science per se, which most scientists also focus on: successful contribution to teaching, the transmission of knowledge and science, training and mentoring of younger scientists, promoting of a field of research. 


Do you think the technique will soon become faster and cheaper to be performed on a wide medical scale?

Yes. There are already signs of progress. Scientists either buy the chips sold by some companies or they order cost-free the plasmids that provide the components of the system (protein and RNA). Then they only need to design the RNA molecule that is part of the CRISPR-Cas9 component to create the tool. It is already cheap and this is why it has spread so quickly to research benches all over the world. A small lab running with limited funds can apply the system. CRISPR-Cas9 gene editing has become very democratic. 


You are popular with the media. Do you enjoy standing in the limelight?

This is not a natural exercise for a scientist. However, I feel that CRISPR-Cas9 is an excellent example of a scientific breakthrough originating from pure basic science to highlight to the public and media: blue sky research. There are no old or obsolete topics – one can discover interesting findings in many fields of research. The main focus of the research in my laboratory is to understand molecular and cellular mechanisms of regulation in human host-bacterial pathogen interactions that could ultimately be exploited for the development of novel therapeutic strategies for the treatment of infectious diseases. My initial interest in the CRISPR-Cas9 story was to study small RNA-mediated mechanisms relevant to the virulence and adaptation of the human pathogen Streptococcus pyogenes. It is a wonderful feeling to have highlighted a mechanism that has such a broad range of applications in biomedicine.


Are there any things that you would have done differently if you had a second chance?

I wish that better support would be provided to scientists in Europe with regard to intellectual property issues and the potential commercial applications of research. The US is very active in the field of intellectual property and commercial benefits of discoveries.  


Was it challenging to start thinking about funding? You managed to collect 18.5 million euros to found CRISPR Therapeutics. 

This was a matter of gathering the right team around me. I was just lucky to know Rodger Novak and Shaun Foy, who have long-term experience and expertise in the biotech, pharma and venture capital world. We cofounded CRISPR Therapeutics, which has raised 89 million Euros in series A and B financing rounds. The operations of the company are now located in London and Cambridge, Massachusetts. I do not have an operational role in the company but advise on the science. 


What will be your topic at the EMBO | EMBL Science & Society Conference?  

I will explain the principle of CRISPR-Cas9 to the audience, and present recent developments and applications of the technology, including potential therapies. I will also highlight some ethical concerns that were recently raised with respect to germ line modifications. In these regards, it is critical that the public, scientists, clinicians, developers and ethical specialists understand the technology and appreciate its great benefits for biotechnology and medicine.


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Tilmann KießlingTilmann Kießling
Head, Communications
T. + 49 160 9019 3839