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2015 Genetics Prize

Emmanuelle Charpentier

As a young girl growing up in the environs of Paris, Emmanuelle Charpentier was encouraged by her father, a park manager, and her mother, working in psychiatry, to explore her own academic interests, which were many. “I was a serious student,” she recalls, “but I was interested in a number of things. I liked pure science and mathematics, but I was also interested in the human sciences — psychology, sociology and philosophy.”  She also remembers her father teaching her the Latin names of many plants. “Maybe this influenced me to pursue science,” she says with a laugh.

Charpentier went to the Pierre and Marie Curie University for her undergraduate and graduate studies, receiving a degree in biochemistry in 1991 and a PhD in microbiology in 1995. When Charpentier announced to her family that she would be doing her graduate research at the Pasteur Institute, her mother wasn’t surprised. “She told me that when I was around 12 years old, I had announced that I would be working there one day,” she recalls. 

The lab felt immediately like home to Charpentier. She realized that being a research scientist would fit the many aspects of her personality — her curiosity, her intellectual drive for knowledge, her enjoyment of communicating that knowledge to others and working as a team, and her desire to turn complex scientific discoveries into practical applications that would help society. “I was excited about being a scientist,” she says. 

At the Pasteur Institute, Charpentier investigated the genetic and molecular mechanisms behind antibiotic resistance. Then, as new molecular and cellular technologies became available in the early 1990s, she turned her focus to the study of how bacterial pathogens infect and interact with their hosts and environment, focusing on the molecular mechanisms during infection with the aim to find new pathways that could be further harnessed for the benefit of medicine. She also developed tools to facilitate the genetics in bacterial pathogens and studied proteins involved in cell-cell adhesion and signal transduction by developing transgenic mouse models. In 1996, she moved to the United States, where during the next five years she held research associate positions at three New York City institutions — the Rockefeller University, New York University’s Langone Medical Center, and the Skirball Institute of Biomolecular Medicine — as well as at St. Jude Children’s Research Hospital in Memphis, Tenn. In 2002, she returned to Europe to establish her own research group at the Max F. Perutz Laboratories of the University of Vienna in Austria where she habilitated in the field of Microbiology. She was then appointed Associate Professor and then Guest Professor at the Laboratory for Molecular Infection Medicine Sweden (MIMS, within the Nordic EMBL Partnership for Molecular Medicine) at Umeå University in Sweden where she habilitated in the field of Medical Microbiology. Since 2013, E. Charpentier is Head of the Department “Regulation in Infection Biology” at the Helmholtz Centre for Infection Research, and Professor at the Medical School of Hannover in Germany. 

While in Vienna, Charpentier became interested in regulatory mechanisms in bacteria that involve small RNA molecules. On her move to Umeå, she developed the CRISPR-Cas9 project. One RNA, tracrRNA, retained the attention of her laboratory. This RNA was located in the vicinity of odd DNA sequences known as CRISPR, or “clustered regularly interspaced short palindromic repeats.” When a virus attacks the bacteria, a “snapshot” of the invader’s genetic identity is stored on the CRISPR sequences and then used to repel the virus should it attack again. CRISPR-Cas systems have evolved extensively in bacteria. No one knew, though, exactly how the process for the so-called type II system (CRISPR-Cas9). In 2011, Charpentier published a paper in Nature that identified the critical role that trans-acting CRISPR RNA (tracrRNA) performs in development of the CRISPR-mediated virus defense.

That same year, while at a conference in Puerto Rico, Charpentier met American structural biologist Jennifer Doudna of the University of California, Berkeley, who was investigating how CRISPR systems worked, but from a structural perspective. They decided to collaborate. “I wanted to have the structure of the Cas9 complex to investigate the possibilities to reduce the system to its minimal and identify the position of all active domains in the structure of the complex. We were complementary,” says Charpentier, “and that made working together, even at such long distances, very enjoyable.” 

The following year, in August 2012, Charpentier and Doudna published a paper in Science that has transformed the field of molecular genetics. They showed that when bacteria are invaded a second time by a virus, the copy of the viral genetic information stored on the CRISPR sequences (expressed as the dual tracrRNA:crRNA) recruits a protein called Cas9, which then seeks out and destroys the viral DNA, essentially by cutting it up. This finding has opened up an entire new and exciting approach to genome editing, for it offers a surgical-like technique for removing or adding DNA at targeted locations. The technology is already being used in many laboratories around the world to help develop new treatments for a wide range of human diseases and genetic disorders. 

Charpentier currently lives in Germany, where she is chair of the Regulation in Infection Biology Department at the Helmholtz Centre for Infection Research and a Professor at the Hannover Medical School. She also remains affiliated with the Laboratory for Molecular Infection Medicine at Umea University, Sweden. Charpentier has received many honors and awards, including the Breakthrough Prize for Life Sciences, the Göran Gustafsson Prize from the Royal Swedish Academy of Sciences, the Dr. Paul Janssen Award, the Louis Jeantet Prize for Medicine, the Ernst Jung Prize for Medicine, the Grand Prix Jean-Pierre Lecocq from the French Academy of Sciences, an Alexander von Humbodt Professorship and the Jacob Heskel Gabbay Award in Biotechnology and Medicine. She is a member of the New York Academy of Sciences, the American Academy of Microbiology and the European Molecular Biology Organization (EMBO).