Mechanism of Action

FUS pathology drives neurodegeneration through a dual mechanism — A Loss of Function in the nucleus and a Gain of Toxic Function in the cytoplasm.

Together, these mechanisms explain FUS’s impact on neuronal metabolism: it removes a key regulator from the nucleus while poisoning the cytoplasm.

FUS Dual Mechanism of Disease

Loss of Function (Nuclear Depletion)

  • DNA repair is impaired

  • Transcriptional control of metabolic and stress-response genes collapses

  • Cells unable to adapt to oxidative or metabolic stress

Gain of Toxic Function (Cytoplasmic Mislocalization)

  • Disrupt mitochondrial metabolism and axonal transport

  • FUS aggregation trap RNA and proteins essential for energy balance

  • Toxicity leads to impaired energy production, synaptic dysfunction, and eventual cell death.

MC16 Mechanism of Action

MitoChem’s therapeutic approach with MC16 is designed to addresses both sides of FUS pathology — restoring its nuclear function and reversing its cytoplasmic toxicity.

By correcting FUS localization, we aim to reactivate mitochondrial metabolism, and to restore vital nuclear functions, enabling neurons to withstand disease stress and survive.