From Bacterial Immunity to Genome Editing: The 2014 Dr. Paul Janssen Award Symposium

From Bacterial Immunity to Genome Editing
Reported by
Alan Dove

Posted November 05, 2014

Presented By

Dr. Paul Janssen Award for Biomedical Research

The New York Academy of Sciences


On September 11, 2014, the New York Academy of Sciences and the Dr. Paul Janssen Award for Biomedical Research held a symposium titled From Bacterial Immunity to Genome Editing to honor this year's awardees, Dr. Emmanuelle Charpentier of the Helmholtz Centre for Infection Research and Umeå University and Dr. Jennifer Doudna of the University of California, Berkeley. The researchers were recognized for their role in understanding and adapting the CRISPR/Cas system for genome editing.

Charpentier and Doudna described their work to elucidate how, in bacteria, RNA molecules transcribed from clustered regularly spaced palindromic repeats (CRISPR) mediate adaptive immunity against viruses and foreign plasmids. They discovered that the dual-RNA structures formed between tracrRNA—a small RNA linked to CRISPR—and CRISPR RNAs guide the CRISPR-associated nuclease Cas9 to degrade invading DNA molecules in a sequence-specific manner. Realizing the potential to exploit this system for genome editing, they showed that dual-RNAs could be engineered as single transcripts to target any DNA sequence of interest.

Laboratories around the world are now using this technique to study human diseases such as cancer and HIV, and biopharmaceutical companies are designing CRISPR/Cas-based strategies for gene therapy and drug discovery. After the acceptance speeches by Charpentier and Doudna, three researchers described their work using this breakthrough method for precise manipulation of genetic information, which holds the promise to revolutionize genomic engineering and gene therapy.

Use the tabs above to find a meeting report and multimedia from this event.

Presentations available from:
Emmanuelle Charpentier, PhD (Helmholtz Centre for Infection Research, Hannover Medical School, Germany; Umeå University, Sweden)
Jennifer Doudna, PhD (University of California, Berkeley; Howard Hughes Medical Institute)
Charles A. Gersbach, PhD (Duke Center for Genomic and Computational Biology)
Luciano A. Marraffini, PhD (The Rockefeller University)
William R. Strohl, PhD (Janssen Research & Development)
Moderator: Craig C. Mello, PhD (University of Massachusetts Medical School; Howard Hughes Medical Institute)

This symposium was made possible with support from

  • The Dr. Paul Janssen Award
  • Johnson and Johnson

Journal Articles

Emmanuelle Charpentier

Charpentier E, Marraffini LA. Harnessing CRISPR-Cas9 immunity for genetic engineering. Curr Opin Microbiol. 2014;19:114-9.

Chylinski K, Le Rhun A, Charpentier E. The tracrRNA and Cas9 families of type II CRISPR-Cas immunity systems. RNA Biol. 2013;10(5):726-37.

Chylinski K, Makarova KS, Charpentier E, Koonin EV. Classification and evolution of type II CRISPR-Cas systems. Nucleic Acids Res. 2014;42(10):6091-105.

Deltcheva E, Chylinski K, Sharma CM, et al. CRISPR RNA maturation by trans-encoded small RNA and host factor RNase III. Nature. 2011;471(7340):602-7.

Jinek M, Chylinski K, Fonfara I, et al. A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science. 2012;337(6096):816-21.

Jennifer Doudna

Hochstrasser ML, Taylor DW, Bhat P, et al. CasA mediates Cas3-catalyzed target degradation during CRISPR RNA-guided interference. Proc Natl Acad Sci U S A. 2014;111(18):6618-23.

Jinek M, Jiang F, Taylor DW, et al. Structures of Cas9 endonucleases reveal RNA-mediated conformational activation. Science. 2014;343(6176):1247997.

Nuñez JK, Kranzusch PJ, Noeske J, et al. Cas1-Cas2 complex formation mediates spacer acquisition during CRISPR-Cas adaptive immunity. Nat Struct Mol Biol. 2014;21(6):528-34.

Charles A. Gersbach

Gersbach CA. Genome engineering: the next genomic revolution. Nat Methods. 2014;11(10):1009-11.

High K, Gregory PD, Gersbach C. CRISPR technology for gene therapy. Nat Med. 2014;20(5):476-7.

Kabadi AM, Gersbach CA. Engineering synthetic TALE and CRISPR/Cas9 transcription factors for regulating gene expression. Methods. 2014;69(2):188-97.

Kabadi AM, Ousterout DG, Hilton IB, Gersbach CA. Multiplex CRISPR/Cas9-based genome engineering from a single lentiviral vector. Nucleic Acids Res. 2014. [Epub ahead of print]

Luciano A. Marraffini

Barrangou R, Marraffini LA. CRISPR-Cas systems: prokaryotes upgrade to adaptive immunity. Mol Cell. 2014;54(2):234-44.

Goldberg GW, Jiang W, Bikard D, Marraffini LA. Conditional tolerance of temperate phages via transcription-dependent CRISPR-Cas targeting. Nature. 2014. [Epub ahead of print]

Hatoum-Aslan A, Marraffini LA. Impact of CRISPR immunity on the emergence and virulence of bacterial pathogens. Curr Opin Microbiol. 2014;17:82-90.

Heler R, Marraffini LA, Bikard D. Adapting to new threats: the generation of memory by CRISPR-Cas immune systems. Mol Microbiol. 2014;93(1):1-9.

William R. Strohl

Kinder M, Greenplate AR, Grugan KD, et al. Engineered protease-resistant antibodies with selectable cell-killing functions. J Biol Chem. 2013;288(43):30843-54.

Raju TS, Strohl WR. Potential therapeutic roles for antibody mixtures. Expert Opin Biol Ther. 2013;13(10):1347-52.

Strohl WR. Antibody discovery: sourcing of monoclonal antibody variable domains. Curr Drug Discov Technol. 2014;11(1):3-19.

Strohl WR. Modern therapeutic antibody drug discovery technologies. Curr Drug Discov Technol. 2014;11(1):1-2.

Featured Speakers

Emmanuelle Charpentier, PhD

Helmholtz Centre for Infection Research, Hannover Medical School, Germany; Umeå University, Sweden
website | publications

Emmanuelle Charpentier studied biochemistry and microbiology at the University Pierre and Marie Curie, France, where she received her PhD in microbiology for research at the Pasteur Institute. She spent five years working in the U.S., where she held research associate positions at The Rockefeller University, NYU Langone Medical Center, the Skirball Institute of Biomolecular Medicine, and St. Jude Children's Research Hospital. Charpentier established her own microbiology research group at the Max F. Perutz Laboratories of the University of Vienna, Austria, and was later recruited to the Laboratory for Molecular Infection Medicine Sweden (MIMS, Swedish Node of the European Molecular Biology Laboratory (EMBL) Partnership for Molecular Medicine) at Umeå University. In 2012 she was appointed professor at Hannover Medical School and head of the Regulation in Infection Biology Department at the Helmholtz Centre for Infection Research. In 2013, she received the Alexander von Humboldt Professorship. She is also a recipient of the Erik K. Fernström Prize and the Göran Gustafsson Prize from the Royal Swedish Academy of Sciences. She is a cofounder of CRISPR Therapeutics.

Jennifer Doudna, PhD

University of California, Berkeley; Howard Hughes Medical Institute
website | publications

Jennifer Doudna conducted her PhD research with Jack Szostak at Harvard University. She pursued postdoctoral research with Tom Cech at the University of Colorado, Boulder, as a Lucille Markey Fellow. Doudna established her first research group at Yale University, where she became a professor and Howard Hughes Medical Institute investigator. She is a faculty member in the Departments of Molecular and Cell Biology and Chemistry at the University of California, Berkeley, the Howard Hughes Medical Institute, and the Lawrence Berkeley National Lab. She is also a member of the National Academy of Sciences, a member of the Institute of Medicine, and a fellow of the American Academy of Arts and Sciences. She is the recipient of the Alan T. Waterman Award from the National Science Foundation and of the Lurie Prize in Biomedical Sciences from the Foundation for the National Institutes of Health. Her laboratory pursues mechanistic understanding of fundamental biological processes involving RNA molecules. Research is focused on bacterial immunity via the CRISPR system, RNA interference in eukaryotes, and translational control logic.


Charles A. Gersbach, PhD

Duke Center for Genomic and Computational Biology
website | publications

Charles A. Gersbach is an assistant professor in the Departments of Biomedical Engineering and Orthopaedic Surgery and the Center for Genomic and Computational Biology at Duke University. He received his PhD in biomedical engineering from the Georgia Institute of Technology and Emory University School of Medicine, focusing on the genetic reprogramming of adult stem cells for musculoskeletal tissue regeneration. He completed postdoctoral training at Scripps Research Institute, engineering synthetic enzymes for targeted genome editing in human cells. His laboratory at Duke University is focused on applying molecular and cellular engineering to applications in gene therapy, regenerative medicine, and basic science. In particular, his research aims to develop new methods to genetically modify genome sequences and cellular gene networks in a precise and targeted manner. He is a recipient of the NIH Director's New Innovator Award, the National Science Foundation Career Award, the Hartwell Foundation Individual Biomedical Research Award, the March of Dimes Basil O'Connor Scholar Award, and the Outstanding New Investigator Award from the American Society of Gene and Cell Therapy.

Luciano A. Marraffini, PhD

The Rockefeller University
website | publications

Luciano A. Marraffini received his PhD from the University of Chicago in 2007, studying bacterial pathogenesis in the laboratory of Dr. Olaf Schneewind. He was a postdoctoral researcher at Northwestern University from 2008 to 2010, where he began to investigate the molecular mechanism of CRISPR/Cas immunity with Dr. Erik Sontheimer. In 2010, he joined The Rockefeller University as assistant professor. His laboratory investigates the molecular mechanisms by which CRISPR/Cas systems provide bacteria and archaea with adaptive immunity against viral and plasmid infections. He is a 2012 Rita Allen Foundation Scholar and a 2011 Searle Scholar. He is the recipient of a 2012 NIH Director's New Innovator Award and a 2010 RNA Society Award.

William R. Strohl, PhD

Janssen Research & Development
website | publications

William R. Strohl received his PhD in microbiology from Louisiana State University and worked as a guest researcher at the German Research Center for Biotechnology. From 1980 to 1997, Strohl was a faculty member in the Department of Microbiology and Program of Biochemistry at Ohio State University, researching natural product biosynthesis and generating novel polyketide natural products using genetic engineering approaches. In 1997, he moved to Merck to lead Natural Products Microbiology. He was appointed leader of Merck Monoclonal Antibody Discovery in 2001. In 2008, Strohl joined Janssen Biologics B.V. (formerly Centocor B.V.) to lead Antibody Drug Discovery and later became vice president of Biologics Research at Janssen Research & Development. In 2013, he became head of the Biotechnology Center of Excellence. Strohl is the author of Therapeutic Antibody Engineering: Current and Future Advances Driving the Strongest Growth Area in the Pharma Industry, published by Woodhead Publishing in 2012.


Craig C. Mello, PhD

University of Massachusetts Medical School; Howard Hughes Medical Institute
website | publications

Craig C. Mello is a Howard Hughes Medical Institute investigator, Blais University Chair in Molecular Medicine, and codirector of the RNA Therapeutics Institute at the University of Massachusetts Medical School. His laboratory uses the nematode C. elegans as a model system to study embryogenesis and gene silencing. His work with Dr. Andrew Fire led to the discovery of RNA interference (RNAi), for which they shared the 2006 Nobel Prize in Physiology or Medicine. They showed that when C. elegans is exposed to double-stranded ribonucleic acid (dsRNA), a molecule that mimics a signature of viral infection, the worm mounts a sequence-specific silencing reaction that interferes with the expression of cognate cellular RNAs. RNAi allows researchers to rapidly knock out the expression of specific genes and thus to define the biological functions of those genes. RNAi also provides a potential therapeutic avenue to silence genes that contribute to disease. Mello's work on RNAi was also recognized with the National Academy of Sciences Molecular Biology Award, the Canadian Gairdner International Award, the Paul Ehrlich and Ludwig Darmstaedter Award, and the 2006 Dr. Paul Janssen Award for Biomedical Research. He is a member of the National Academy of Sciences, the American Academy of Arts and Sciences, and the American Philosophical Society.

Alan Dove

Alan Dove is a science writer and reporter for Nature Medicine, Nature Biotechnology, and Bioscience Technology. He also teaches at the NYU School of Journalism and blogs at http://dovdox.com.


This symposium was made possible with support from

  • The Dr. Paul Janssen Award
  • Johnson and Johnson