FTY720 (fingolimod) is a neuroprotective and disease-modifying agent in cellular and mouse models of Huntington disease
2014
alternative therapeutic approach for the treatment of the disease. Here, we utilized preclinical target validation paradigms and examined the in vivo efficacy of chronic administration of FTY720 in R6/2 HD mouse model. Our findings indicate that FTY720 improved motor function, prolonged survival and reduced brain atrophy in R6/2 mice. The beneficial effect of FTY720 administration was associated with a significant strengthening of neuronal activity and connectivity and, with reduction of mutant huntingtin aggregates, and it was also paralleledbyincreasedphosphorylationofmutanthuntingtin atserine13/16 residuesthatarepredicted toattenuate protein toxicity.
Keywords:
- Correction
- Source
- Cite
- Save
- Machine Reading By IdeaReader
62
References
68
Citations
NaN
KQI