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FTD Basic Mechanisms: Characterize TDP-43 and FUS roles in FTD (Milestone 2.P)

In Progress

Timeline Start - End

2016 - 2025

Research Implementation Area

Research on Disease Mechanisms

AD-Related Dementias Focus

Determine the mechanism of TDP-43 and FUS pathogenesis and toxicity.

Success Criteria

  • Support research to identify the predominant mechanism(s) of TDP-43 and FUS-related pathogenesis and neurodegeneration in FTD.

  • Determine if and how aggregated TDP-43 spreads.

Summary of Key Accomplishments

Abnormal clumps of the proteins TDP-43 and FUS are a hallmark of FTD, ALS and related neurodegenerative diseases. Ongoing NIH-funded projects are providing new insights into how these aggregates form. For example, recent studies have shown that TDP-43 forms droplets inside cells that are prone to turn into a gel or solid during times of cellular stress. Other studies have shown that defects in the cell nucleus lead to TDP-43 and FUS dysfunction, ultimately resulting in neuronal death. Novel possible targets for therapeutic intervention in FTD and FTD-ALS are emerging from this research.

The key accomplishments summary is current as of July 2022.  

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