Abstract
Sangamo BioSciences, Inc., (CA, USA) is the pioneer and leader in the next phase of molecular medicine; genome editing. The company's proprietary platform is based on a human DNA-binding motif, the zinc finger DNA-binding protein (ZFP). Sangamo engineers ZFPs to bind to virtually any DNA sequence with exquisite specificity, making the platform uniquely suitable for therapeutic applications. Zinc finger nucleases can be designed to enable therapeutic genome editing, to knockout a gene or add a DNA sequence to an investigator-chosen site in the genome. The company is focused on developing novel ZFP Therapeutics(®) as potentially curative therapies for genetic and infectious diseases, including hemophilia, lysosomal storage disorders, hemoglobinopathies and HIV/AIDS. Sangamo is the only company with human clinical trials of this novel technology.
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