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FTD Basic Mechanisms: Understand role of genetic mutations (Milestone 2.O)

AD Related Dementias Focus

Please note: This milestone was incorporated into Milestone 2-N in 2019.

Determine the molecular basis for C9ORF72 expansion-and GRN mutation-related neurodegeneration.


Success Criteria

  • Identification of predominant mechanism(s) of C9ORF72 and GRN mutation-related FTD/ALS pathogenesis, with convergent findings in human tissues and model systems.

  • Prioritization of targets to move forward into therapy development after testing therapeutic hypotheses in model systems.


Research Implementation Area
Research on Disease Mechanisms
Timeline
2016–2025
Status
In Progress

Accomplishments/Implementation Activities

Funding Initiatives

Relevant Recommendations

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