La Jolla Institute Opens Major RNAi Center For Identyfing Genetic Triggers Of Disease

August 12, 2011 -- A major Center that will propel scientific efforts to pinpoint the specific genes involved in causing immune diseases, cancer and other diseases will be opened today at the La Jolla Institute for Allergy & Immunology. Utilizing a Nobel prize-winning technology known as RNA interference (RNAi), the Institute's new RNAi Center will be a catalyst for accelerating discovery toward new therapies against myriad diseases, and is one of a small, select group of dedicated RNAi facilities worldwide.

"Today's opening of our RNAi Center represents a milestone for fueling research on the genetic basis of diseases," said Mitchell Kronenberg, Ph.D., the La Jolla Institute's president and chief scientific officer, and the RNAi Center's co-principal investigator with Anjana Rao, Ph.D., a prominent genetics and cell biology researcher recruited from Harvard Medical School last year. "Our Center will focus the collective talents of an exemplary group of RNAi researchers on understanding the genetics behind disease processes of all kinds, and will use that knowledge toward developing new therapies to treat disease."

The Center, to open today with a formal dedication ceremony, was funded through a $12.6 million grant from the National Institutes of Health (NIH) under the American Recovery and Reinvestment Act of 2009 (Recovery Act), and is designed to be a collaborative, openly available resource for the scientific community. "The RNAi Center will be open to scientists at academic research institutions on the Torrey Pines Mesa and around the country and is welcome news to our colleagues at Salk, Scripps, and UC San Diego, who wrote letters of support for our RNAi grant proposal," said Dr. Kronenberg. The Scripps Research Institute is already involved, with two of its scientists, David Nemazee and Changchun Xiao, Ph.D.s, to lead one of the Center's first four projects.

The RNAi Center's four initial projects were funded as part of the NIH grant and will draw on the La Jolla Institute's immunology expertise. The projects aim to make key discoveries about how the body recognizes bacteria and viruses and fights infections. The projects also aim to understand how the immune system can sometimes hurt the body, and what genes cause these problems, which underlie the development of autoimmune diseases like multiple sclerosis, type 1 diabetes and rheumatoid arthritis. These immune system diseases are areas for which the La Jolla Institute's scientific research is particularly renowned.

"We're going to use this great technology to make breakthroughs in biomedical research, along with making the Center, and our RNAi experts, available to the wonderful talent at our fellow San Diego research institutions," said Dr. Kronenberg, adding that a primary NIH goal in funding the Center was to boost genetic research due to its strong potential for improving human health.

RNAi has been heralded as a revolutionary technology because it opens the door to developing new therapies for cancer and other diseases based on silencing specific genes. Its discoverers were awarded the 2006 Nobel Prize for Physiology or Medicine.

Sonia Sharma, Ph.D., the RNAi Center's scientific director, said that the type of research to be performed at the Center will enable the next great leap in the understanding of gene functions in health and disease. "The Human Genome Project, completed in 2003, gave us the genetic sequence of the 20,000 to 30,000 genes that make up the genetic blueprint of every individual," she said. "This was an enormous advance, but in some ways like receiving a book, where you can see the words on the page, but don't necessarily understand the true meaning of each word," she said.

"RNAi lets us explore the function of each gene, so that we can determine how it fits into the disease process," added Dr. Sharma. Using RNAi, researchers can shut off individual genes, one at a time, in order to figure out which functions they control. Once medical researchers know, for instance, that a certain gene is a major contributor to a specific disease process, they can make it a target for future drug development, she explained.

Based on the earliest RNAi research, clinical trials have already been initiated in the U.S. for several diseases, including hepatitis C and leukemia. The technology is also currently being used to explore macular degeneration, a major cause of blindness in the elderly.

Dr. Kronenberg said RNAi advances thus far are promising and that the Institute's specialized expertise in real-life experimental models of disease will enhance the capabilities of RNAi even further. "We intend to develop efficient methods for RNAi screening in living organisms, which will allow us to analyze complex disease processes like Alzheimer's or cancer in ways never before possible," he said. "Large-scale screening using mouse models will be revolutionary because it will greatly expand the current capabilities of RNAi."

The new Center will feature the latest in high-throughput automation and robotics, combined with extensive libraries of RNAi molecules. "We will use advanced RNAi technology to screen against a subset of human genes, or even against the entire human genome, to determine the effect on a disease process," said Stephen Wilson, Ph.D., Center executive director and the Institute's VP/Chief Technology Officer.

"Our Center's strong capabilities will enable La Jolla Institute researchers, and others around the country, to meet the challenge of sifting through thousands of genes in a relatively short time to pinpoint the key genetic players in human disease," he said.

In addition to Recovery Act funding from the NIH Office of the Director, the La Jolla Institute RNAi Center is supported by the National Institute of Allergy and Infectious Diseases, part of the NIH.