Sigma-Aldrich scientists to leverage proprietary gene editing technology to develop genetically-engineered rodent research models

Aug 24 2009

Sigma-Aldrich(R) (Nasdaq: SIAL) announced today that scientists at its new Sigma Advanced Genetic Engineering (SAGE(TM)) Labs in St. Louis will leverage its exclusive CompoZr(TM) Zinc Finger Nuclease (ZFN) gene editing technology (www.compozrzfn.com) to define, develop and offer a new generation of genetically-engineered rodent research models. Such models are commonly used to study disease states in humans and play a key role in the drug discovery process.

Employing methods used to generate the world's first rat with a targeted gene deletion, commonly referred to as a 'Knockout' as introduced in the July 24, 2009 issue of the Journal Science(R), "Knockout Rats via Embryo Microinjection of Zinc-Finger Nucleases", scientists at SAGE Labs will initially create Knockout rat models with focus in toxicology, neuroscience, cardiovascular and inflammatory disease, areas that would benefit most immediately from the promise represented by the availability of the Knockout Rat. Using this novel methodology, Knockout Rats and Mice can be generated in as little as four months, about one-third the time required to make a Knockout model using conventional Embryonic Stem Cell (ES cell) based approaches.

"The Knockout Rat is a long-awaited milestone in the scientific community, and we are pleased that Sigma innovation has played such a key role in its development. Applying our proprietary CompoZr ZFN technology in this very practical application has provided researchers with a powerful new tool to study human diseases. The potential applications using these rodent models of human disease could ultimately eliminate years of research time and save millions of lives," said Dr. Dave Smoller, President, Sigma Research Biotechnology.

For over a quarter century researchers have been trying to generate Knockout rats that closely mimic human diseases, a breakthrough now possible due to the gene-editing capabilities of ZFN technology, which is significant, as rats are highly characterized animals and physiologically similar to humans. Because their larger size makes them superior to mice in biological research applications, rats are ideal subjects for research into human diseases.