ion of animals in which a chosen gene is specifically removed or "knocked out" from the pig's genome. In the published example, Sangamo scientists and their collaborators in the laboratory of Prof. Dr. Heiner Niemann of the Institute of Farm Animal Genetics in Germany, knocked out both copies of the 1,3-galactosyl transferase (GGTA-1
) gene from pig cells and used these to produce cloned animals that lack the target gene. Knockout of the GGTA-1
gene has been shown to lead to significantly improved organ survival in a pig-to-baboon organ transplantation model.
"Sangamo's mission is to develop novel ZFP Therapeutics to address unmet medical needs and make paradigm-shifting therapeutic solutions a reality," stated Edward Lanphier, Sangamo's president and chief executive officer. "This proof of concept study lays the foundation for the use of our validated ZFP platform technology to modify animal organs for human transplantation."
Efficient Generation of a Biallelic Knockout in Pigs using Zinc-Finger Nucleases.
Hauschild J, Petersen B, Santiago Y, Queisser AL, Carnwath JW, Lucas-Hahn A, Zhang L, Meng X, Gregory PD, Schwinzer R, Cost GJ, Niemann H.
Proc Natl Acad Sci U S A. 2011 108 (29) 12013-12017.
Sangamo BioSciences, Inc. is focused on research and development of novel DNA-binding proteins for therapeutic gene regulation and modification. The most advanced ZFP Therapeutic® development program is currently in a Phase 2b clinical trial for evaluation of safety and clinical effect in patients with diabetic neuropathy. Sangamo also has a Phase 1 / 2 clinical trial and two ongoing Phase 1 clinical trials to evaluate the safety and efficacy for the treatment of HIV/AIDS as well as a Phase 1 trial for the treatment for recurrent glioblastoma multiforme. Other therapeutic programs are focused on Parkinson's disease, monogenic diseases, neuropathic pain and nerve regeneratio
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