Scientists are excited by somewhat unusual things. For example, I am not that interested in meeting movie stars, but when I met the astronaut Jim Lovell I was speechless (the club of people who have been in space is pretty small). Therefore, I was delighted to be invited to the ceremony for surely what is one of many awards that will be bestowed upon discovers of CRISPR/Cas9 genome engineering, Dr. Jennifer Doudna and Dr. Emmanuelle Charpentier. I was joined by Addgene Scientist Matt Ferenc (one of our resident CRISPR experts) at the 2014 Johnson & Johnson’s Dr. Paul Janssen Award dinner which took place at the New York Public library. Being in the presence of scientific heroines is always inspiring. I was especially excited to attend this event because Jennifer did her graduate work one floor above mine in the lab of Dr. Jack Szostak, who went on to win the Nobel Prize. Dr. Doudna has had a host of other impressive mentors including Tom Cech, Robert Tijian, Tom Steitz and Joan Steitz.
This award ceremony honored Charpentier and Doudna, who talked about their research leading up to the discovery of the mechanism of dual-RNA-guided DNA cleavage in adaptive bacterial immunity and its potential applications in rewriting the genome. Their award acceptance addresses were inspiring! We also got to hear more from them during a panel discussion with Dr. Craig Mello and Dr. Paul Stoffels from J&J. The panel was moderated by a non-scientist from Bloomberg news. Given the topic it might have been better to have a scientist up there, but the panelists were interesting enough to make up for the somewhat naïve questions. During the panel, an artist was sketching her interpretation of the discussion in markers on a whiteboard. The final image is pictured here…she started from a blank white slate and developed this over 30 minutes, which was fun to watch.
A Simpler 1 Protein - 1 RNA System
Drs. Dounda and Charpentier met at a 2011 conference and decided to collaborate on what was a “curiosity” driven project. Both awardees emphasized the contribution of students in their labs and the importance of the strong, cross-disciplinary collaboration they shared to the work they did together. They especially thanked Martin Jinek and Krzysztof Chylinski (who coincidentally speak the same Polish sub-dialect making their Skype communications extra fun) who shared first authorship on their seminal, cooperative paper in Science. Interestingly, this project started with their labs studying the mechanisms of RNAi, the field for which Craig Mello and Andrew Fire won the Nobel Prize. What did Dr. Doudna think was the most important part of their work? First, the discovery of a small, programmable DNA cutting enzyme. Second, since gRNA and crRNA could be combined into one RNA to direct cleavage the result was a simpler 1 protein - 1 RNA system.
Dr. Charpentier advocated for the crucial role of basic research in the scientific community and the importance of this type of experimentation to the eventual translation to the clinic and cures. There is no doubt that companies are swarming to see what can be done with this technology to help patients. Dr. Stoffels and others in the audience discussed how J&J is already modifying cell and animal genomes to test hypotheses of disease. They are finding they have been able to use CRISPR/Cas9 to generate mice with genes knocked out in the insulin signaling cascades in only 4 months compared to a year or greater with older technologies. Addgene is proud to be helping get CRISPR reagents out to academic labs. It is our hope that robust, open academic exploration in this area will accelerate the launch of clinical applications.
During the panel discussion, Dr. Mello took it upon himself to explain the science to the non-scientists in the room. He offered the great analogy of comparing the CRISPR/Cas9 system to Norton Antivirus. Antivirus programs work by taking info from the code of viruses to give information to the computer to fight the known viruses. Bacteria have figured out how to download a bit of virus sequence to enable them to recognize and fight it forever. He emphasized the potential of this extraordinary breakthrough. In fact, the panelists agreed that, for example, the technology is already available today to cure sickle cell anemia in stem cells but this can’t be used in people until we learn more about safety and more about the how to use genome edited stem cells to treat disease.
As for me, I continue to be amazed at how cool biology can be and how much we have yet to learn. As Dr. Mello said, “Cells have been in the information age for billions of years (…) Bacteria are really sophisticated”.
New to CRISPR/Cas9 Technology? Learn More:
- Addgene's CRISPR/Cas9 Guide
- History of CRISPR Cas - A tale of survival and evolution
- A Powerful New Way to Edit DNA (by Andrew Pollack of New York Times)