Session Abstract – PMWC 2017 Silicon Valley

Session Synopsis: Since its launch, the disruptive CRISPR technology has been widely adopted and is now on a path to transform biological research with the promise of new therapeutics for both treating and curing human diseases. This session will cover new CRISPR products and different applications that harness the power of the CRISPR technology.

Session Chair Profile

Director of Advanced Genomics, MilliporeSigma

Dr. Shawn Shafer has been at MilliporeSigma for 7 years and currently serves as the Director of Advanced Genomics. His group oversees the development of molecular tools for genetic research and diagnostic applications. As the leader of this team, Shawn oversaw the creation of MilliporeSigma’s comprehensive portfolio of CRISPR products, with a focus on targeted genome editing in cells and research animals, as well as high throughput screening applications. Dr. Shafer received his PhD in Developmental Biology from Washington University and began his science career working on the Human Genome Project. Leading a sequencing lab on this project gave Shawn a taste for “big” science, with many people working towards a common goal. At the same time, Dr. Shafer witnessed the value of academic and industry partnerships in facilitating big science and has worked to bring the two together so that researchers on either side have access to the best technology.


High Throughput Knockout Screening with CRISPR
The adoption of the CRISPR pathway components for targeted genetic engineering has received much attention, but CRISPR has also fundamentally changed how we approach screening. In this talk we will discuss the important considerations to make for high throughput knockout screening with CRISPR in various formats and review the accompanying data. We will conclude with a look toward the future of screening and the promise that these new tools hold for uncovering previously recalcitrant targets.

Speaker Profile

Ph.D., Professor of Chemistry and Molecular and Cell Biology, University of California, Berkeley

Jennifer Doudna, Ph.D. helped launch an ongoing revolution in the fields of molecular genetics and genomics with the monumental discovery of CRISPR-Cas9. This simple to use technique can alter the DNA of any organism using RNA-programmed DNA cleavage, much like a film editor cuts a piece of film and splices in new frames. CRISPR-Cas9 technology is being used in laboratories around the world to advance biological research and this fundamental technology promises to lead to new therapeutics for treating and curing, human disease. This is just the latest chapter in a highly productive career of scientific discovery. Doudna has devoted her career to understanding the function of catalytic and other non-protein-coding RNAs. Using structural biology and biochemistry, Doudna’s work deciphering the molecular structures and biochemical activities of RNA enzymes (ribozymes) and other functional RNAs, along with their protein-binding partners, has shown how these molecules carry out complex activities in cells. Dr. Doudna has been an investigator with the Howard Hughes Medical Institute since 1997. In 2000, while holding a professorship at Yale, she was honored with the Alan T. Waterman Award, given annually by the National Science Foundation to an exceptional young scientist. In 2002 she accepted a faculty position at University of California, Berkeley where CRISPR-Cas9 was discovered. Jennifer Doudna earned her Bachelor of Arts degree in Chemistry from Pomona College in 1985, her Ph.D. in biochemistry from Harvard University, and she conducted her postdoctoral work at the University of Colorado, Boulder.


The Genome Engineering Revolution
Facile genome manipulation using precision DNA recognition is transforming biology. I will discuss how the bacterial CRISPR adaptive immune system was harnessed as a powerful genome engineering tool, enabling remarkable developments using this technology to modify, regulate or visualize genes in a wide variety of cells and organisms.

Speaker Profile

Ph.D., Investigator, Howard Hughes Medical Institute; Professor, Cellular & Molecular Pharmacology, University of California, San Francisco

Jonathan Weissman, PhD studies how cells ensure that proteins fold into their correct shape, as well as the role of protein misfolding in disease and normal physiology. He is also widely recognized for building innovative tools for broadly exploring organizational principles of biological systems. These include ribosome profiling, which globally monitors protein translation, and CRIPSRi/a for controlling the expression of human genes and rewiring the epigenome. Dr. Weissman is a professor at the University of California San Francisco and an Investigator at the Howard Hughes Medical Institute. He is a member of the National Academy of Sciences, a member of Scientific Advisory Board for Amgen and co-director of the Innovative Genome Initiative of Berkeley and UCSF. Dr. Weissman has received numerous award including the Beverly and Raymond Sackler International Prize in Biophysics (2008), The Keith Porter Award Lecture from the American Society of Cell Biology (2015) and the National Academy Science Award for Scientific Discovery (2015).


Controlling Gene Expression with CRISPRi and CRISPRa
We have developed a robust technology, based on catalytically dead CRIPSR-Cas9 variants, for reversibly turning on (CRISPRa) or off (CRISPRi) the expression of any gene. I will discuss applications of CRISPRi/a for understanding disease mechanisms, defining drug targets, and treating disease by reversibly regulating gene expression without permanently altering patients’ DNA.