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Emmanuelle Charpentier and Jennifer Doudna Share $500,000 Gruber Genetics Prize for Landmark Discovery of RNA-Guided CRISPR/Cas9 Editing System; Prize to Be Awarded Friday, October 9, at ASHG Annual Meeting

The 2015 Gruber Genetics Prize will be awarded this year to microbiologist Emmanuelle Charpentier, Ph.D., of the Helmholtz Centre for Infection Research in Braunschweig, Germany, and biochemist Jennifer Doudna, Ph.D., of the University of California, Berkeley. These two eminent scientists are being recognized for their joint creation of a revolutionary gene-editing technology known as CRISPR/Cas9, which functions as a molecular scissor, generating double-stranded cuts in targeted DNA molecules with exceptional precision. The technology is being used around the world to advance biological research and to engineer genes for developing powerful new therapies for a wide range of human diseases, as well as new biofuels and agricultural products. The award will be presented to Dr. Charpentier and Dr. Doudna in Baltimore, Maryland, on Friday, October 9, during the 2015 annual meeting of the American Society of Human Genetics (ASHG). “The discovery of the CRISPR/Cas9 cellular defense system has transformed molecular genetics,” said Utpal Banerjee, Ph.D., a member of the Selection Advisory Board to the Prize. Dr. Banerjee is Professor and Chair of the Department of Molecular, Cell, and Developmental Biology at UCLA. He did his post-doctoral fellowship in the laboratory of the renowned Seymour Benzer, Ph.D. “We now have a quick and highly accurate technology for deleting or adding specific pieces of DNA, an advance with wide-ranging implications for both basic science and clinical medicine,” Dr. Banerjee added. Dr. Charpentier and Dr. Doudna began their collaboration in 2011 after meeting at a scientific conference in Puerto Rico. Both had been trying to unlock the molecular mysteries of the CRISPR systems, an unusual repeating sequence of DNA that enables bacteria to mount a successful defense against viral invaders.

Dr. Charpentier was focusing on the type II system (CRISPR/Cas9) and had described, in a seminal paper published in Nature in 2011, the three components of the system that consist of two RNAs forming a duplex (tracrRNA and crRNA) and the protein Cas9 (formerly named Csn1) and the roles of each component in the early steps of activation of the system.

A year later, in their seminal 2012 paper in Science, Dr. Charpentier and Dr. Doudna showed that an RNA-guided protein, Cas9, “reads” genetic information on CRISPR sequences and then uses that information to seek out and destroy the invading viral DNA, essentially by cutting it up.

The two scientists also demonstrated that this cellular defense system had applications beyond killing viruses, for the RNA could be engineered to attach to any gene, thus encouraging the Cas9 protein to cleave at that spot. That made the CRISPR system an exceptionally powerful tool, for it could be customized to delete or add specific strands of DNA.

“Drs. Charpentier and Doudna have made an extraordinary contribution to molecular biology and genetics,” says Huda Zoghbi, M.D., Chair of the Selection Advisory Board to the Prize. Dr. Zoghbi is Professor in the Departments of Pediatrics, Molecular and Human Genetics, Neuroscience, and Neurology at Baylor College of Medicine; and she is also a Howard Hughes Medical Institute (HHMI) Investigator.

“Their research has led to an entire new approach to genome editing, which promises to open up new strategies for biological studies in many diverse organisms and for potentially treating diseases as varied as HIV/AIDs, sickle cell anemia, and muscular dystrophy. It’s a great honor to be awarding them with this prestigious award.”

In addition to the cash award, each recipient will receive a gold laureate pin and a citation that reads as follows.

“The Gruber Foundation proudly presents the 2015 Genetics Prize to Emmanuelle Charpentier and Jennifer Doudna for establishing a framework for universal genome editing. Charpentier and Doudna discovered that the bacterial enzyme Cas9 is an endonuclease that cuts DNA at sites specified by a guide RNA, and defined biochemically the components required for this reaction. They showed that the sequence of the guide RNA could be modified to target the endonuclease to virtually any site. This provided the mechanism by which bacteria acquire immunity to specific viral infections, allowed introduction of specific mutations at desired sites, and provided the means to transfer efficient Cas9-directed break, repair, and editing to any cell type. This method has broadly enabled genome editing for uses in basic biology, medicine, biotechnology, and agriculture.

[Gruber Foundation press release] [Nature 2011 article] [Science 2012 abstract]