Improve motor function
in Double KO mice
Not tested Not tested Yes Yes Yes Yes Yes
Halt axonal degeneration
in Double KO mice
Not tested Not tested Yes Yes Yes Yes Yes
Biochemical Changes in ABCD1- Mice Reduced VLCFA levels in brain and spinal cord Reduced VLCFA levels in various tissues and CNS Normalize oxidative stress,
Prevent mitochondria depletion
Restore redox homeostasis,
stimulate mitochondrial biogenesis
Reduce oxidative damages,
Prevent mitochondria depletion
Normalize ER stress Restore Autophagy,
prevent accumulation of oxidized proteins
Mechanism/
Targeted pathway
Gene regulation Upregulate ABCD2 expression Mitochondria biogenesis Regulate energy metabolism Antioxidant pathway ER stress Autophagy dysfunction
Treatment AAV Thyromimetics Pioglitazone High-dose biotin DMF TUDCA Temsirolimus