Prizewinners have radically simplified genome editing and engineering
Emmanuelle Charpentier and Jennifer A. Doudna have developed the bacterial gene cutter CRISPRCas9
in such a way that specific DNA sequences can be targeted and cleaved. The technology is
opening up completely new perspectives for research.
Emmanuelle Charpentier from France and Jennifer A. Doudna from the
USA were awarded the Paul Ehrlich and Ludwig Darmstaedter Prize for 2016 in the
Paulskirche in Frankfurt. The two scientists were being honored for their pioneering work in the
development of the programmable gene editing tool CRISPR-Cas9. "With this precision tool genes
can be modified easily and precisely," wrote the Scientific Council of the Paul Ehrlich Foundation in
explaining its decision. "The prizewinners recognized and identified this potential and demonstrated
the technology's far-reaching applications." In the shortest time, CRISPR-Cas9 has become one of the
most sought-after tools in molecular biology research, the Scientific Council continues. CRISPR-Cas9
is so easy to use that genome editing, which only a few years ago was extremely complicated, has
become a routine procedure. The Council also paid tribute to the fact that Doudna and Charpentier
were among the first to call for debate on the ethical issues because CRISPR-Cas9 can also be used to
edit and engineer the germline. Charpentier is Director at the Max Planck Institute of Infection
Biology in Berlin and Professor at Umeå University in Sweden. Doudna is the Li Ka Shing
Chancellor's Professor at the University of California, Berkeley. The Paul Ehrlich and Ludwig
Darmstaedter Prize is among the most prestigious international awards granted in the Federal Republic
of Germany in the field of medicine. The Prize was presented by Professor Harald zur Hausen,
Chairman of the Scientific Council.
Charpentier and Doudna were the first to demonstrate that CRISPR-Cas9, developed by bacteria as a
defense against bacteriophages, can be used to target and cleave any DNA sequence. The gene cutter
is programmed and controlled by a guide RNA. One of the prizewinners' achievements is that they
have made the gene cutter easier and more user-friendly. Experiments in numerous laboratories have
quickly shown that this simplified form works not only in the test tube but also in living cells and in
many organisms. The DNA is edited and engineered in repairing double strand breaks. This makes it
possible to replace or alter genes or knock them out of action. There is strong evidence that CRISPRCas9
will help cure hereditary diseases, eradicate dangerous pathogens, and breed better plants.
The two prizewinners' pioneering publication in 2012 - following the breakthrough finding by
Charpentier in 2011 of the CRISPR-Cas9 system - unleashed a veritable wave of CRISPR-Cas9
research. Since then, thousands of publications have appeared that reveal its true potential and describe
further details and potential developments of the CRISPR technology. "This technology is changing
both fundamental research and clinical and commercial opportunities in biology," says Doudna.
"That's very exciting." Charpentier says: "I think that CRISPR-Cas9 has the potential to really change
the biotechnology and the medical landscapes."
At the ethics summit held in Washington in December 2015, both scientists spoke out against editing
of the human germline for clinical applications at this time. In the concluding statement, which
Doudna as one of the organizers also signed, germline editing is deemed to be "irresponsible" unless
and until the relevant ethical and safety issues have been resolved. The document also declares that
norms concerning acceptable uses should be established by the international community. The summit
did not call for a moratorium but rather for intensification of research within the legal and ethical
boundaries.
Short biography of Professor Dr. Emmanuelle Charpentier
Emmanuelle Charpentier was born in Juvisy-sur-Orge, France, in 1968. She studied
microbiology, genetics and biochemistry in Paris and completed her doctorate at the Institut Pasteur.
After working in New York and Memphis, notably at the Rockefeller University, Charpentier moved
to the University of Vienna in 2002 and to Umeå University in 2009, where she is still Visiting
Professor. Charpentier came to Germany in 2013 on a Humboldt Professorship. She first headed a
Department at the Helmholtz Center for Infection Research in Braunschweig and was full Professor at
the Hannover Medical School. She was appointed Director at the Max Planck Institute for Infection
Biology in Berlin in October 2015. Charpentier has been awarded well over two dozen different
prizes, including the three million dollar Breakthrough Prize in Life Sciences, the Leibniz Prize of the
German Research Foundation and an honorary doctorate of the University of Louvain. She is a
member of various science academies and associations, including Germany's Academy of Sciences
Leopoldina and elected foreign member of the Royal Swedish Academy of Sciences. She co-founded
CRISPR Therapeutics in 2014 and ERS Genomics in 2013.
Short biography of Professor Dr. Jennifer A. Doudna
Jennifer A. Doudna was born in Washington, DC and grew up in Hilo, Hawaii. She studied
chemistry at Pomona College in California and took her doctorate in biological chemistry and
molecular chemistry at the Harvard University in 1989. After her doctorate, Doudna moved to the
University of Colorado. In 1994 she was appointed professor at Yale, and she has been a professor at
the University of California, Berkeley, since 2002. At Berkeley she serves as Chair of the Chancellor's
Advisory Committee on Biology. Doudna is also the Executive Director of the Innovative Genomics
Initiative at UC Berkeley/UCSF. The co-prizewinner has been an investigator with the Howard
Hughes Medical Institute since 1997. She has won numerous awards, including the three million dollar
Breakthrough Prize in Life Sciences. Doudna is a member of various science academies and
associations, including the National Academy of Sciences, the National Academy of Medicine, the
National Academy of Inventors and the American Academy of Arts and Sciences. She is also a cofounder