Drug Compound Halts Alzheimer’s-related Damage in Mice

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Appears to revers some neurological harm

Under ordinary circumstances, the protein tau contributes to the normal, healthy functioning of brain neurons. In some people, though, it collects into toxic tangles that damage brain cells. Such tangles are a hallmark of Alzheimer’s and other neurodegenerative diseases. But researchers at Washington University School of Medicine in St. Louis have shown that levels of the tau protein can be reduced – and some of the neurological damage caused by tau even reversed ­– by a synthetic molecule that targets the genetic instructions for building tau before the protein is made. The findings suggest that the molecule – known as an antisense oligonucleotide – potentially could treat neurodegenerative diseases characterized by abnormal tau, including Alzheimer’s. “We’ve shown that this molecule lowers levels of the tau protein, preventing and, in some cases, reversing the neurological damage,” said Timothy Miller, MD, PhD, the David Clayson Professor of Neurology and the study’s senior author. “This compound is the first that has been shown to reverse tau-related damage to the brain that also has the potential to be used as a therapeutic in people.”

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