Sangamo’s technology (ZFP) for efficient, permanent correction of disease-causing gene accepted for publication by Nature

Sangamo BioSciences, Inc. (Nasdaq: SGMO) today announced publication of data that demonstrates the use of the Company’s zinc finger DNA-binding protein (ZFP) technology to achieve highly efficient, permanent correction of a disease-causing gene in primary human cells. This research, published in Nature as an advance online publication, represents a significant advance in the ability to specifically and efficiently modify the human genome and provides the scientific foundation for potential therapeutic approaches for a variety of genetic disorders and infectious diseases.

“For years, scientists have been searching for a way to modify or edit the genome of plants and animals in a precise and predictable fashion,” said Nobel Laureate, Professor Sir Aaron Klug, of the MRC Laboratory of Molecular Biology, Cambridge, UK. “This work is therefore truly a landmark study that provides the foundation for gene modification-based therapeutics without the safety issues that have plagued many traditional gene therapy applications. It gives me great personal satisfaction to see this remarkable outcome of my original discovery of zinc fingers and their development.”

For gene modification, ZFPs are being used in combination with a DNA cutting enzyme (endonuclease) functional domain to generate ZFNs that facilitate the correction of mutant gene sequences that cause disease or the disruption of genes that facilitate disease progression.

Source: Sangamo BioSciences