Kavli Workshop will highlight powerful genome-editing tool, CRISPR-Cas9

The gene-editing tool known as CRISPR-Cas9 is taking science by storm. This Thursday, at a workshop organized by the Kavli Institute for Neuroscience, a handful of Yale scientists will discuss how they are using the powerful new technique to solve diverse problems in biology and medicine.

The workshop is geared at Yale researchers who are interested in adding the new technique to their toolboxes. The five speakers, including organizer Shawn Ferguson, an assistant professor of cell biology at the Yale School of Medicine, will discuss how they are putting CRISPR-Cas9 to work in their laboratories. They include Yale neuroscientists Ferguson and Susumu Tomita, geneticists Jun Lu and Sidi Chen, and Timothy Nottoli, who co-directs the Yale Genome Editing Center, which offers a range of gene-editing services.

"This workshop is an opportunity for a lot of labs to get up to speed on what CRISPR-Cas9 has to offer,” says Ferguson, who is using CRISPR-Cas9 to study the location of certain proteins within cells. “My lab has used these new genome-editing tools for several years now and we have seen that they are powerful for a variety of different purposes, including disruption of gene function and the tagging of proteins to allow visualization of their subcellular localization."

CRISPR-Cas9 is based on a system used by bacteria to fend off viruses and other threats. It consists of several key molecules: a nuclease enzyme called Cas9 and RNAs that guide Cas9 toward specific DNA sequences in the genome. The result is a RNA-protein complex that snips strands of DNA at a precise location. Cellular DNA repair machinery can subsequently stitch the cut DNA back together again. This molecular cut-and-sew method works in most organisms, including humans.

Gene editing techniques based on CRISPR/Cas9 are having a transformational impact in biology and neuroscience, as they make gene manipulation accessible, practical and rapid

Harnessing Cas9 for use in mammalian cells has revolutionized the ability of scientists to understand the role of different genes and to manipulate their activity. It far outshines other tools by giving scientists a fast, accurate and inexpensive way to delete, modify or insert genes.

“Gene editing techniques based on CRISPR/Cas9 are having a transformational impact in biology and neuroscience, as they make gene manipulation accessible, practical and rapid,” says Pietro De Camilli, director of the Kavli Institute for Neuroscience and chair of the Department of Neuroscience at the Yale School of Medicine. “The goal of this workshop is to bring together Yale investigators who have already developed a major expertise with this methodology to share their experience with the neuroscience community at large.”

While Cas9 evolved as part of a bacteria anti-viral defense strategy, it has been adapted for use in eukaryotic cells by the efforts of several labs, including those of Jennifer Doudna (University of California, Berkeley), Emmanuelle Charpentier (Max Planck Institute for Infection Biology, Berlin), Feng Zhang (MIT) and George Church (Harvard University).

The workshop will be held in the Hope 110 conference room from 1:30 to 5:30 pm on Thursday, June 23, 2016. For more information, visit: kavli.yale.edu/events/