Sangamo Biosciences Inc (SGMO)

[surf1944]

7:07AM Sangamo BioSci announces first presentation of data from ZFP Therapeutic for Huntington's Disease at 2012 annual meeting of society for neuroscience; novel zfp therapeutic approach selectively represses only the disease-causing gene (SGMO) 5.85 : The data demonstrate that a ZFP Therapeutic, based on Sangamo's zinc finger DNA-binding protein (ZFP) gene regulation technology, can selectively repress the expression of the mutant disease-causing form of the huntingtin gene (HTT) while leaving expression levels of the normal gene unchanged in cells derived from HD patients. In multiple independent cell lines derived from HD patients carrying different, disease-causing CAG repeat lengths, they demonstrated that these ZFP TFs decreased production of the mutant HTT messenger RNA (mRNA) by >90% while leaving the levels of the normal HTT mRNA largely unchanged (a reduction of 10% or less); in turn, this achieved similar selective reduction in levels of mutant compared to normal HTT protein. Furthermore, in neurons derived from a HD mouse model (R6/2), ZFP TFs selectively repressed the expression of mutant HTT, which is required for disease expression in these animals. In strong support of the safety of this approach, an unbiased genome-wide expression analysis confirmed the exquisite specificity of the ZFP TFs targeted to the HTT CAG-repeat alone.