EFFICIENT NEUROPROTECTIVE RESCUE OF SACSIN-RELATED DISEASE PHENOTYPES IN ZEBRAFISH

The fish model of Autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS) (Sacs−/− larvae) was treated with Acetyl-DL-leucine. Acetyl-DL- leucine improved locomotor and biochemical phenotypes in disorder by mediating significant rescue of the molecular functions altered by sacsin loss, demonstrating the neuroprotective effect of the treatment. International Journal of Molecular Sciences. 

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