RNA Interference Library Covers Entire Human Genome

Provides Biotech & Pharma Industry Powerful New Tool

Cold Spring Harbor, NY, March 24, 2004 - In a paper published this week in the journal Nature (March 25), scientists at Cold Spring Harbor Laboratory announced the creation of the first library of human RNA interference (RNAi) clones, which will eventually provide users the ability to shut off virtually every gene in the human genome. Based on a versatile and powerful method for triggering RNAi - short hairpin RNA (shRNA) - the library is the first DNA vector-based, human genome-wide RNAi library that is sequence verified.

Several leading pharmaceutical companies have entered into agreements with Long Island-based Cold Spring Harbor Laboratory (CSHL), to take advantage of the strong potential for therapeutic discovery that this powerful library promises to deliver.  Licensing non-exclusive rights allows these companies to use the RNAi library for target identification and validation purposes.

"The beauty of the RNA interference phenomenon is its potential to shut off individual genes and only those genes," said Dr. Greg Hannon, lead scientist in the development of the new library.  "By individually targeting more than 10,000 human genes through this sequence-based method, a wide variety of companies can rapidly identify and validate target genes that cause disease, and develop drugs to hit those targets."

RNA interference is sweeping the biotech world, because it provides researchers a convenient way to silence individual genes.  A variety of methods exist for triggering RNAi.  However, the short-hairpin (shRNA) method developed by Hannon and his colleagues is one of the most efficient and has been validated in a large number of studies with different animal and human cell cultures as well as in whole animals, where the method has been shown by Hannon's group to trigger stable, heritable gene silencing.  In addition, the sequence-validated library of short hairpin RNA molecules targets each one of more than 10,000 different human genes in triplicate, i.e. with three different gene-specific interfering RNAs.

"We hope that the pharmaceutical and biotechnology industries will use our method to systematically search for targets for new drugs for cancer and other diseases," said Hannon.

Cold Spring Harbor Laboratory is a recognized leader in the field of RNA interference research.  In 2002, Hannon's work with his CSHL partners  was named "Breakthrough of the Year" by the journal Science, which cited its potential for drug discovery and a variety of other applications.  Although it is used widely in the research world as a genetic tool, RNAi's potential in the commercial world is just now being realized.

"We are proud to be at the forefront of this phenomenon, and are extremely excited by the interest that the biotech and pharmaceutical sectors are beginning to take in the vast potential of the new library" said John Maroney, Director of Technology Transfer at Cold Spring Harbor Laboratory.  "We believe that this method is one of the most powerful and accurate ways for any company to identify targets and develop drugs based on the human genome sequence."  Maroney is currently in discussions with several companies seeking to license the library and accompanying technology.  "Industry leaders now recognize that they are at a significant disadvantage without robust RNAi technology at their disposal."

For information on access to the RNAi library created at Cold Spring Harbor, please contact the Office of Technology Transfer at (516) 367-8301.

Cold Spring Harbor Laboratory is a private, non-profit basic research and educational institution. Under the leadership of Dr. Bruce Stillman, a member of the National Academy of Sciences and a Fellow of the Royal Society (London), more than 300 scientists conduct groundbreaking research in cancer, neurobiology, plant genetics and bioinformatics.