Sigma-Aldrich Launches Breakthrough Genome-Editing Tools

Sigma-Aldrich has launched its CompoZr™ platform of zinc finger nuclease (ZFN) reagents. The CompoZr ZFN platform of reagents is expected to provide researchers with the ability to target and manipulate the genome of living cells, resulting in cell lines or whole organisms with defined gene deletions, insertions, or corrections.

Initially offered as a customized service for developing ZFNs for specific gene targets, the CompoZr ZFN platform will eventually include ZFN-based kits for targeted transgene insertion and a catalog of off-the-shelf reagents for commonly studied gene targets, gene families, and pathways.

Applications for Sigma's new platform technology include creation of knockout and knock-in cell lines, cell lines that yield significantly greater amounts of protein, and novel transgenic animal models.

ZFN reagents are a class of engineered DNA-binding proteins that facilitate targeted editing of the genome within a living cell by creating double-strand breaks in DNA at user-specified locations. Double-strand breaks stimulate the cell's natural DNA-repair processes, namely homologous recombination and non-homologous end joining, to induce site-specific mutagenesis.

Using CompoZr reagents, researchers can, for the first time, generate targeted genomic edits using a single-step procedure, resulting in cell lines with permanent and heritable gene deletions, insertions, or modifications.

Additionally, Sigma-Aldrich is collaborating with leading academic researchers to demonstrate the effectiveness of ZFNs in the creation of whole animal knockout models in mice and other species, including rats and rabbits.

"Zinc Finger Nuclease technology has held significant promise for more than a decade and is now mature enough to fundamentally alter the way in which research on living cells and organisms is conducted," said Dr. David Smoller, President of Sigma-Aldrich's Research Biotech Business Unit. "By refining ZFN production methods and utilizing our established distribution capabilities, Sigma-Aldrich is making this exciting technology available to life science researchers worldwide."

By facilitating the creation of complete gene knockout and knock-in somatic, embryonic and primary cell lines, ZFNs allow researchers to precisely determine the biological function of a gene in more relevant backgrounds. It is anticipated that ZFN-mediated genome editing could be used to generate novel animal models of disease that more closely mimic human disease, as well as providing more realistic data on the potential toxicity of new drug compounds.

For biopharmaceutical manufacturers, ZFNs will enable the creation of cell lines with improved growth characteristics, altered glycosylation properties, and other traits resulting in higher yields.

Through its exclusive partnership with Sangamo BioSciences, Inc., Sigma Life Science is the sole source of validated research reagents incorporating ZFN technology. Sigma Life Science employs high-throughput production methods to create customized ZFN reagents for user-specified gene targets in just six to eight weeks.

All CompoZr reagents are functionally validated in a cell line appropriate for the intended organism of interest, and customers are provided with full protocols for use of their reagents and troubleshooting support from Sigma Life Science's experienced technical team.