Sangamo BioSciences and Sigma-Aldrich Announce Nature Biotechnology Study Demonstrating Zinc Finger Technology for Modification of Human ...(pair and the ability to efficiently add ...),Health

Date:10/29/2007

pair and the ability to efficiently add a therapeutic gene into the genome of a cell at a predetermined location or to correct a mutated gene in a patient's stem cells may enable us to provide a long term solution for many genetic diseases. The powerful combination of our highly efficient delivery platform and Sangamo's ZFN technology made possible unprecedented gene modification efficiency in these therapeutically important cells".

The data published in the Nature Biotechnology paper demonstrate that ZFN-mediated gene modification can be used to correct both mutations in the IL-2R gamma gene, the defective gene in X-linked severe combined immunodeficiency (X-linked SCID), and to add a therapeutic gene to a pre-determined 'safe-harbor' site in the genome of both human hematopoietic stem cells and human embryonic stem (ES) cells. This putative 'safe-harbor site' was selected by the investigators for its capacity to allow efficient expression of the therapeutic gene and to tolerate an insertion event without adverse effects.

"These data open new avenues for experimental biology, biotechnology and medicine," said Philip Gregory, D.Phil., Sangamo's vice president of research. "This year's Nobel Prize for Medicine recognized the importance of gene targeting, or the site specific manipulation of the mouse genome, which has revolutionized biology and enabled the generation of mouse cell-lines and transgenic mouse models of human disease. However, until now and the development of our ZFN technology, genomes of other species such as humans and plants could not be efficiently modified in a site specific manner. Our ZFN technology enables the specific and targeted modification of any genome of potentially any species and may enable the development not only of potential therapeutics but also human cell lines and transgenic animals bedsides the mouse that may be valuable models of human disease for medical research and drug development."

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SOURCE Sangamo BioSciences, Inc. Copyright©2007 PR Newswire. All rights reserved

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