2014 Dr. Paul Janssen Award Symposium Celebrates CRISPR Genome Editing Pioneers

NEW YORK, September 4, 2014 - Described as one of the most significant breakthroughs in molecular biology in the past decade, discovery of the dual-RNA-guided DNA cleavage mechanism in adaptive bacterial immunity has opened-up extraordinary potential for rewriting the genome, and is likely to drive innovation and new developments in human disease modelling and treatment of genetic disorders.  

The discovery was made by this year's Dr. Paul Janssen Award for Biomedical Research winners; Emmanuelle Charpentier PhD, of the Hannover Medical School and Helmholtz Centre for Infection Research, Germany, and The Laboratory for Molecular Infection Medicine Sweden, Umeå University, Sweden and Dr. Jennifer Doudna PhD of the University of California, Berkeley and Howard Hughes Medical Institute. The researchers will be honored at the event, From Bacterial Immunity to Genome Editing: The 2014 Dr. Paul Janssen Award Symposium, on September 11, 2014, presented by the Dr. Paul Janssen Award for Biomedical Research and the New York Academy of Sciences.

Charpentier and Doudna began their research trying to understand how, in bacteria, RNA molecules transcribed from Clustered Regularly Spaced Palindromic Repeats (CRISPR) facilitates adaptive immunity against viruses and foreign plasmids. They realized that CRISPR-encoded RNAs form dual-RNA structures that guide the CRISPR-associated nuclease Cas9 to degrade invading DNA molecules in a sequence-specific manner. This realization allowed them to show that dual-RNAs could be engineered as single transcripts to target any DNA sequence.

"The CRISPR-Cas9 molecular tool is easy to use and can easily be designed to modify genes and correct mutations. It has already proven its potential in genetic manipulation of various organisms and cells. The tool also allows us to move forward our understanding of gene functions, human gene therapy, and regenerative medicine" says Emmanuelle Charpentier PhD.

 "This discovery could not have come about without collaboration. Collaboration in science is becoming more and more important as knowledge advances. Scientists with different types of expertise are able to come together and solve problems that any one of us would find difficult to do individually," says Jennifer Doudna PhD. She adds, "The celebration of scientific achievement is important inspiration for young people who are considering a career in research; they love to see that discoveries that solve problems in society are recognized by both other scientists and the community at large."

Following Award lectures by Charpentier and Doundna, leading scientists in microbiology, molecular genetics, and genomics will discuss the implications of this new technology for basic science, medical research, drug development, and human health. Speakers will include:

• Charles A. Gersbach, PhD, Duke University
• Luciano A. Marraffini, PhD, The Rockefeller University
• William R. Strohl, PhD, Janssen Research & Development, LLC

All speakers will participate in a wrap-up panel discussion on "the Transformative Power of Basic Research for Bioengineering and Biomedicine" moderated by Craig C. Mello, PhD, University of Massachusetts Medical School and Howard Hughes Medical Institute.

Symposium registration is free, made possible by support from Johnson & Johnson and the Dr. Paul Janssen Award for Biomedical Research. For more information visit www.nyas.org/Janssen2014.

For press inquiries, including press passes to the conference, please contact Stacy-Ann Ashley (sashley@nyas.org; 212-298-8696).

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