New CRISPR/Cas9 Innovative Genomics Initiative

The University of California, Berkeley, and UC San Francisco are launching the Innovative Genomics Initiative (IGI) to lead a revolution in genetic engineering based on a new technology already generating novel strategies for gene therapy and the genetic study of disease.

The Li Ka Shing Foundation has provided a $10 million gift to support the initiative, establishing the Li Ka Shing Center for Genomic Engineering and an affiliated faculty chair at UC Berkeley. The two universities also will provide $2 million in start-up funds.

A new genomic engineering technology significantly cuts down the time it takes researchers to test new therapies. CRISPR/Cas 9 allows the creation in weeks rather than years of animal strains that mimic a human disease, allowing researchers to test new therapies. The technique also makes it quick and easy to knock out genes in human cells or in animals to determine their function, which will speed the identification of new drug targets for diseases.

In the past, for example, making a strain of mice with a specific and heritable genetic mutation took at least a year of costly experiments. Using the Cas9 technique, UC Berkeley immunologist Russell Vance disabled a gene in mice that regulates fur color and in just six weeks had a strain of mice with white coats instead of brown. Similar research in animal models ranging from rodents to primates is being done in labs around the world using the CRISPR/Cas9 technology.

Other researchers have already adapted the technology to reprogram stem cells to regenerate damaged organs, such as the liver, and made attempts to reprogram immune cells to cure AIDS in HIV-positive patients.

Doudna and her colleagues also are building resources and infrastructure for an incubator to assist researchers, postdoctoral fellows and students to spin off companies. This Entrepreneurial Fellows program will coordinate with the QB3 Startup-in-a-Box program to help launch new companies that address important societal challenges and create new jobs in California.

The Cas9 technology adapts a DNA-snipping system used by bacteria to cut up and destroy the DNA of invading viruses. Doudna and colleagues Martin Jinek of the Howard Hughes Medical Institute at UC Berkeley and Emmanuelle Charpentier of the Helmholtz Centre for Infection Research (and formerly of the Laboratory for Molecular Infection Medicine-Sweden) discovered how the enzyme Cas9 works with small RNA molecules to target and cut specific areas of DNA. Jinek and Doudna also showed that the RNA-programmable CRISPR/Cas9 system works in human cells, and is much faster and easier than other genome editing methods.

Initiative codirector Michael Botchan, UC Berkeley professor of molecular and cell biology, is most excited about the potential to explore the 95 percent of the human genome once called “junk DNA” that is now known to contain most of the regulators that turn genes on and off, and may harbor defective genes responsible for many diseases.

“What makes humans different from monkeys is not the 6-7 percent of the genome that codes for proteins, but the regulatory genes in the rest of the genome,” he said. “But we don’t know what most of these regulatory genes do. It’s likely they are involved in diseases like schizophrenia, diabetes and many others.”

The CRISPR/Cas9 technology also allows researchers to disrupt multiple genes or regulatory sequences at once to determine their function and interactions. This kind of research is likely to uncover new targets for drugs, Botchan said.

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