• Gene Scissors to Combat Hereditary Diseases

    Molecular biologist Professor Emmanuelle Charpentier receives the Hansen Family Award 2015

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    Revolutionizing biology: Professor Emmanuelle Charpentier has developed a method that is now used in laboratories all over the world.

As one of the most innovative scientists in the field of molecular biology, Professor Emmanuelle Charpentier has ­developed a kind of molecular scissors that enables targeted editing of the genome. In recognition of this achievement, she was awarded the 2015 Hansen Family Award.

Bacteria take an uncompromising approach to intruders – they simply shred the threat. If foreign viruses introduce their genome into the bacteria, for example, their genetic scissors get straight to work, snipping the unwanted genome back out and cutting it into pieces to render it harmless. This defense mechanism developed by the bacteria, known as the CRISPR-Cas9 system, intrigued French biologist Professor Emmanuelle Charpentier (46), who ultimately unraveled the details of the mechanism. “My team and I are researching the mechanisms of infections from the perspective of bacteria. How they survive, how they adapt, protect themselves, multiply and, ultimately, cause diseases.” The microorganisms intentionally integrate parts of the viruses’ DNA into their own genome, thus strengthening their immunity against other attackers.

Charpentier recognized the potential this tool used by bacteria could offer molecular biology right from an early stage. She and her team therefore decoded the bacterial protection system. Together with the group led by Professor Jennifer Doudna, a researcher and teacher at the University of California, she also demons­trated that the cutting mechanism could be reproduced and used in different ways, including genome editing. This process is of particular interest to many other researchers around the world who are working to separate genome strands at specific points, remove certain gene sections or add new ones, and make corrections or replacements in order to, for example, add new traits to certain plant types or develop gene therapies. Until recently, this was truly a Sisyphean task, requiring months of effort on the scientists’ part. “I wanted to turn the bacteria’s genetic scissors into a universal tool as a means of simplifying and speeding up my fellow scientists’ work,” says the biologist.

Prize-winning: Kemal Malik, member of the Bayer AG Board of Management responsible for innovation, congratulates Professor Emmanuelle Charpentier on her successful research.

Bacteria the Models for Genetic Engineers Worldwide

And, together with Doudna’s team, she sensationally managed to find a way of getting the CRISPR-Cas9 system to work in the lab as an RNA programmable genetic editing tool. These achievements origi­nating from basic science earned Charpentier the 2015 Hansen Family Award worth EUR 75,000, which is awarded by the Bayer Science & Education Foundation for pioneering research in innovative fields of biology and medicine. Using the same method as the bacteria, the CRISPR-Cas9 system enables the DNA strand to be cut at precisely defined points – allowing ­scientists to repair genes, for example. Every gene contains a blueprint for protein molecules, each of which performs a specific function. However, if the genetic code contains a fault, this produces a defective protein – and can cause hereditary disea­ses. But the new technology developed by Charpentier now enables researchers to precisely cut out the defective genes and replace them with the correct elements. “It’s comparable with swapping one word for another in a text on a computer,” explains Charpentier, who foresees applications for this tool in medicine and other fields of research. “The greatest potential surely lies in using this technology to treat hereditary diseases, such as cystic fibrosis or sickle-cell anemia,” she believes.

Bayer Alumni Dialogue 2015

Charpentier developed a keen interest in biology early on, and it was already her favorite subject at school. She went on to study biochemistry and microbiology at the Université Pierre & Marie Curie in Paris and has conducted research at a number of universities in the United States. She qualified as a professor of microbiology in 2006. Today, Charpentier is well known as a pioneer and one of the most innovative researchers in the field of molecular-biological infection research. Following teaching posts at the Helmholtz Center for Infection Research in Braunschweig and Hannover Medical School, Charpentier was appointed Director of the Berlin-based Max Planck Institute for Infection Biology in October 2015. She is also Visiting Professor at Umeå University where she developed the CRISPR-Cas9 research.

My team and I are researching the mechanisms of infections from the perspective of bacteria. How they survive, how they adapt, protect themselves, multiply and, ultimately, cause diseases

The CRISPR-Cas9 system developed by Charpentier and her team now provides researchers with a tool to locate defective gene sequences, remove them and insert healthy sections.

Hope for New Therapeutic Approaches for Hereditary Diseases

“What’s special about CRISPR-Cas9 is that it is so simple,” Charpentier explains. “You could call it a pair of target-seeking ­molecular scissors. The technology is already being used worldwide as a molecular biology tool for developing new thera­pies for hereditary and chronic diseases.”

The Bayer Foundations – Committed to Progress Since 1897

Bayer foundations have been promoting education, science and social innovation all over the world since 1897. As part of the innovation company Bayer, the foundations see themselves above all as initiators, promoters and partners for progress at the interface between industry, science and the social sector. Their programs are focused on pioneers – their commitment to public welfare, their wealth of ideas in resolving social tasks, and their creativity in the fields of science and medicine. The Bayer Science & Education Foundation, for example, grants scholarships and awards which encourage young talents and top researchers alike to deliver outstanding achievements in their field. The Bayer foundations also
support efforts to resolve social issues. For example, the Bayer Cares Foundation focuses on citizens’ projects and resolving issues in the field of social medicine. The objective of the foundations is always to improve human life through innovation and initiatives.

Health camp – young people explore medical careers

Insights into Health Care Professions

In the “Fascination health – how does it work?” educational project, schoolchildren receive an insight into health care careers. The project weeks are initiated and supported financially by the Bayer Science & Education Foundation.

The day before yesterday it was drug researchers, yesterday emergency doctors, today it’s nurses, and tomorrow geriatric nurses – every day the schoolchildren are able to discover a whole new career in the health care sector. For a whole week, they had the opportunity to gather hands-on experience of the various aspects of these jobs. “Our aim is to nurture interest among these young people in the whole range of careers in the health care sector,” says Jürgen Möller, who accompanied the project at training provider Provadis. The concept for the project week, which was held in Frankfurt in July 2015, was developed by Provadis Partner für Bildung und Beratung GmbH. A second project week followed in October. Entitled “Fascination health – how does it work?”, the project enabled a group of 14- to 16-year-olds from a variety of school types to experience a number of roles. As chemists, for example, Bayer trainees helped them to manufacture acetylsalicylic acid – the active ingredient in Aspirin® – and then check the quality of what they had produced. As well as in the biology and chemistry laboratories at Provadis, the children also experienced work at Frankfurt’s health authority, the geriatric nursing school in Hufeland-Haus and the nursing school including intensive care at the Rotkreuz hospital in July camp as well as the hospital “zum heiligen Geist” in October camp. Provadis works with a number of cooperation partners to provide the schoolchildren with this experience. Along with the institutions already mentioned, the project included eight high schools in the Frankfurt area and the German Federal Employment Agency. Says Möller, “It enables the young people to come into contact with institutions and careers that they would not normally see at their age. As a result, they can think now about whether they would like to work in the health care sector in the future.”iated and supported financially by the Bayer Science & Education Foundation.

Source: Provadis, 2015