Award
National Institute on Aging 1K22AG086336-01
Identifying and characterizing new therapy targets in TDP-43 proteinopathies
Recipient
Washington University
Award Amount
$162,000.00
Ceiling
$162,000.00
Awarded
February 27, 2026
Identifier
1K22AG086336-01
This award funds research to identify and characterize new therapeutic targets in TDP-43 proteinopathies, which are linked to neurodegenerative diseases such as ALS, FTD, and AD. The study focuses on the role of ATXN3 APA and TDP-43 in RNA metabolism to develop potential therapies.
Description
The project aims to determine the functional significance of ATXN3 APA and identify pathways to modulate its expression in human neurons. Additionally, it will explore TDP-43's role in RNA metabolism, identify new TDP-43 target genes, and develop cellular tools to study TDP-43 nuclear clearance. Therapies targeting TDP-43 dysfunction could impact a broad group of neurodegenerative disease patients. Understanding the mechanisms of TDP-43 mislocalization and identifying modifiers like ATXN3 and MARK3 could lead to novel therapeutic strategies.