A world-renowned scientist, Dr. Charpentier is widely recognized for her seminal research contributions to the discovery and development of the CRISPR (clustered regularly interspaced short palindromic repeats)-Cas9 (CRISPR associated protein 9) gene-editing system. This system and Dr. Charpentier ‘s ongoing research continues to contribute to groundbreaking progress in the fields of molecular biology and genetics.
Her most notable research discoveries stem from her investigations involving CRISPR-mediated viral immunity in the pathogenic bacterium, Streptococcus pyogenes. In these studies, she characterized the various components of the system, which conveys resistance to recurrent viral infections. She described how the process first requires duplexing of CRISPR RNA (crRNA) and trans-activating crRNA (tracrRNA) molecules and showed that tracrRNA is involved in the maturation of crRNA. She subsequently showed that these two RNAs lead to the recruitment of the RNA-guided DNA endonuclease, Cas9, and induce double-strand DNA (dsDNA) cleavage at target DNA sequences, work that was reported in a study published with Dr. Jennifer Doudna and other colleagues.
Drs. Charpentier, Doudna, and colleagues further modified the CRISPR system by joining the crRNA:tracrRNA duplexes together to create a versatile technique for gene editing that utilizes single-guide RNAs (sgRNAs). Together they proved that sgRNAs can be utilized to add or delete select DNA sequences by directing Cas9-mediated DNA breaks at target sequences. This system has and continues to revolutionize numerous fields of biomedical research, including cancer, by providing a flexible tool by which to repair genetic defects. In addition to its use in cancer, the CRISPR-Cas9 system is being investigated for the treatment of diseases that are linked to specific gene mutations, such as Duchenne muscular dystrophy and cystic fibrosis, as well as for various agricultural uses that improve disease resistance in crops.
2017 Albany Medical Center Prize in Medicine and Biomedical Research, Albany, New York
2017 Elected Foreign Associate, National Academy of Sciences
2017 Japan Prize
2016 Gottfried Wilhelm Leibniz Prize, German Research Foundation
2016 L’Oréal-UNESCO Award for Women in Science
2016 Otto Warburg Medal
2015 Breakthrough Prize in Life Sciences
2015 Carus Medal, German National Academy of Sciences Leopoldina
2015 Ernst Jung Prize for Medicine
2015 Louis-Jeantet Prize for Medicine, Geneva, Switzerland
2015 Peter and Patricia Gruber Foundation Award in Genetics
2015 Princess of Asturias Award for Technical and Scientific Research
2014 Dr. Paul Janssen Award for Biomedical Research
2014 Göran Gustafsson Prize in Molecular Biology, Royal Swedish Academy of Sciences
2014 Grand Prix Jean-Pierre Lecocq, French Academy of Sciences
2011 Eric K. Fernström Prize, Umeå University, Sweden
2009 Theodor Körner Prize, Vienna, Austria