Sangamo Presents Dramatic Data Indicating Their ZFP Enzymatic Approach Can Prevent and Reverse Pathogenic Accumulation of Mutant Protein in Huntington’s Disease in Animal Models; Wexler Terms Results a “Significant Step Forward”

On November 19, 2014, Sangamo BioSciences, Inc., announced its presentation of positive preclinical data from its joint program with Shire plc, to develop a novel ZFP (zinc-finger protein) Therapeutic® approach to Huntington's disease (HD), at the 2014 Annual Meeting of the Society for Neuroscience. Note that zinc fingers are proteins that normally bind at different bases in DNA in order to regulate gene activity. The dramatically exciting data were generated by Sangamo scientists and the CHDI Foundation, which is dedicated to accelerating therapeutic developments for HD. "These data are very exciting and represent a significant step forward in the quest for a therapeutic for Huntington's disease," said Nancy Wexler, Ph.D., Higgins Professor of Neuropsychology in the Departments of Neurology and Psychiatry of the College of Physicians and Surgeons at Columbia University, and the President of the Hereditary Disease Foundation. "They provide the first demonstration of a therapeutic approach that can not only prevent, but reverse the accumulation of mutant Huntingtin protein aggregates in the brains of animal models of the disease. Furthermore, the treatment does not affect the expression of the normal form of the protein, which is believed to be essential." The mutant form of the Huntingtin protein (Htt) accumulates in cells and forms protein aggregates which are associated with disease symptoms. Pioneering basic research in transgenic animal models has shown that the levels of the defective Htt protein correlate with disease progression, stimulating the search for strategies to reduce mutant Htt levels as a therapeutic intervention. However, most "Htt-lowering" methods decrease the levels of both disease-causing and normal forms of Htt.
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