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How Ionis’ antisense drugs could help combat ALS and dementia – FierceBiotech

Scientists at Mayo Clinic and the University of Pennsylvania shed light on the role of a protein called poly(GR) in toxic TDP-43 aggregation, a hallmark of frontotemporal dementia and amyotrophic lateral sclerosis. They showed in mice that antisense drugs by …

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Frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS) are two fatal neurodegenerative diseases marked by different clinical signs. But they are believed to be caused by a common underlying mechanism: a mutation in the C9orf72 gene that leads to the toxic accumulation of the TDP-43 protein.
Now, scientists at Mayo Clinic and the University of Pennsylvania are providing additional insight into the problem by shedding light on the role of another protein called poly(GR) in TDP-43 a…

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