A 30,000 Foot View of TDP43 Direct Targeting Strategies – A Thought Experiment
- Overview of TDP-43 pathology as the central disease hallmark in the majority of ALS patients
- Current therapeutic approaches targeting TDP-43 loss of nuclear function
- Strategies aimed at mitigating toxic gain of function, aggregation, and cytoplasmic mislocalization
- Key lessons from the field, remaining translational challenges, and future directions for TDP-43–targeted therapies