Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced the publication of a preclinical study demonstrating highly specific, functional correction of the alpha 1-antitrypsin (A1AT) gene defect in patient-derived induced pluripotent stem cells (iPSCs) using zinc finger nucleases (ZFNs). The study, published in Nature, further highlights the precision and broad applicability of ZFN-based genome-editing for the development of ZFP Therapeutics® for the treatment of monogenic diseases.
"These data demonstrate the potential of combining human iPSCs with ZFN-driven gene correction to generate differentiated cell-based therapies," stated Philip Gregory, D. Phil., Sangamo's vice president of research and chief scientific officer. "Importantly, analysis of the entire coding sequence of a ZFN-corrected iPSC line revealed that the only modification attributable to ZFN activity was the intended repair of the A1AT gene. This demonstrates the singular specificity that can be achieved using Sangamo's ZFP technology."
The work was carried out in collaboration with scientists at the Wellcome Trust Sanger Institute, the University of Cambridge, U.K., DNAVEC Corporation of Japan, and several laboratories in Europe. The study was published as an Advance Online Publication in Nature on October 12, 2011 (http://www.nature.com/nature/journal/vaop/ncurrent/full/nature10424.html).
"Our precise ZFN genome-editing technology enables Sangamo to modify any target gene of interest," said Edward Lanphier, Sangamo's president and chief executive officer. "As this study demonstrates, our platform can generate novel ZFP Therapeutic options for diseases that have a well-defined genetic cause. In addition to our ZFN clinical programs in HIV, addressing unmet medical needs such as hemophilia and other monogenic diseases is a focus of our therapeutic pipeline."