Rational design of small molecules targeting TDP-43 liquid-to-solid phase transition for ALS/FTD treatment

🧪 Hypothesis Details

TDP-43 undergoes pathological liquid-liquid phase separation in ALS/FTD, and small molecules that restore liquid-like properties could theoretically address both nuclear loss-of-function and cytoplasmic gain-of-function toxicity. However, this hypothesis faces fundamental barriers: no defined binding pocket exists for intrinsically disordered regions, distinguishing pathological from physiological LLPS remains unsolved, and no validated biomarker exists for target engagement. Precedent failure o...

This artifact has no version history yet.

↻ Rerun processing chain

No linked artifacts recorded yet.