RNA Interference Library Covers Entire Human Genome
Provides Biotech & Pharma Industry Powerful New Tool
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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.
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