New DNA-editing technology spawns bold UC initiative

BERKELEY

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.

Jennifer Doudna, executive director of the new Innovative Genomics Initiative and the new Li Ka Shing Chancellors Chair in Biomedical and Health Sciences.

At the core of the initiative is a revolutionary technology discovered two years ago at UC Berkeley by Jennifer A. Doudna, executive director of the initiative and the new faculty chair. The technology, precision DNA scissors referred to as CRISPR/Cas9, has exploded in popularity since it was first published in June 2012 and is at the heart of at least three start-ups and several heavily-attended international meetings. Scientists have referred to it as the holy grail of genetic engineering and a jaw-dropping breakthrough in the fight against genetic disease. In honor of her discovery and earlier work on RNA, Doudna received last month the Lurie Prize of the Foundation for the National Institutes of Health.

Professor Doudnas breakthrough discovery in genomic editing is leading us into a new era of possibilities that we could have never before imagined, said Li Ka-shing, chairman of the Li Ka Shing Foundation. It is a great privilege for my foundation to engage with two world-class public institutions to launch the Innovative Genomics Initiative in this quest for the holy grail to fight genetic diseases.

In the 18 months since the discovery of this technology was announced, more than 125 papers have been published based on the technique. Worldwide, researchers are using Cas9 to investigate the genetic roots of problems as diverse as sickle cell anemia, diabetes, cystic fibrosis, AIDS and depression in hopes of finding new drug targets. Others are adapting the technology to reengineer yeast to produce biofuels and wheat to resist pests and drought.

We now have a very easy, very fast and very efficient technique for rewriting the genome, which allows us to do experiments that have been impossible before, said Doudna, a professor of molecular and cell biology in the California Institute for Quantitative Biosciences (QB3) and an investigator in the Howard Hughes Medical Institute at UC Berkeley. We are grateful to Mr. Li Ka-shing for his support of our initiative, which will propel ground-breaking advances in genomic engineering.

Transforming genetic research The 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.

The CRISPR/Cas9 technology is a complete game changer, said Jonathan Weissman, codirector of the initiative and professor of cellular and molecular pharmacology in the UCSF School of Medicine. With CRISPR, we can now turn genes off or on at will. I am particularly interested in using CRISPR to understand the normal functions of genes as well as how disease-causing mutations alter these functions.

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New DNA-editing technology spawns bold UC initiative