Sangamo’s Gene Regulation Technology Reduces Alzheimer’s-linked Tau Protein

Sangamo’s Gene Regulation Technology Reduces Alzheimer’s-linked Tau Protein
Sangamo Therapeutics' gene regulation technology significantly reduced levels of tau – a protein crucial to Alzheimer’s disease (AD) – in laboratory tissue experiments and a mouse model, the company announced. Sangamo's collaborators from the Massachusetts General Hospital Alzheimer's Disease Research Center and Harvard Medical School presented the results at the 13th International Conference on Alzheimer's & Parkinson's Diseases in Vienna. In addition to extracellular amyloid plaque, accumulation of tau protein and neurofibrillary tangles in the brain are hallmarks of Alzheimer's. The tangles ultimately lead to neuronal dysfunction and loss. Reducing tau levels protects neurons and may reverse the disease, research has shown. "Tau protein plays a critical role in certain devastating neurodegenerative diseases, including dementias such as AD, and studies point to the potential for tau reduction to prevent neuronal loss and possibly even reverse Alzheimer's disease pathology," Bradley Hyman, MD, PhD, said in a press release. Hyman is the director of the Massachusetts Alzheimer's Disease Research Center and of the Alzheimer's Unit at the MassGeneral Institute for Neurodegenerative Disease. He is also a neurology professor at Harvard Medical School. The study involved Sangamo's zinc finger protein transcription factor (ZFP-TF)-mediated gene regulation technology. ZFP-TF selectively represses or activates the expression of a specific gene or gene sequence. That makes it different from approaches such as genome editing or gene therapy, which aim to correct or replace a missing or mutated gene. Importantly, ZPF-TF can be applied to a broad range of diseases. "Of the many approaches to reduce tau expression that we've studied, zinc finger protein
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