ARSACS is incurable neurodegenerative disorder and there is an urgent need to define new therapies. The expected results of this project are to evaluate, through preclinical studies, the effectiveness and usefulness of an experimental diet containing neuroprotective factors – acetyl-DL leucine (ADLL) and tauroursodeoxycholic acid (TUDCA) – to improving the condition of sacs adult mutant zebrafish. The experimental diets will be administered to zebrafish from the fifth day post-fertilization (when the larvae start feeding independently) until adult life. During their development, zebrafish will be monitored for growth parameters, locomotor performances, social and cognitive behavior. The overall goal of this proposal is to offer a valuable tool to identify compounds that could act on the proper development of the nervous system and able to rescue the pathological phenotypes of zebrafish ARSACS model.

Grant: $40,000

Duration: one year


Dr. Valentina Naef

Stella Maris Foundation (IRCCS)

via dei Giacinti 2 56128 Calambrone, Pisa Italy