Modulating the phosphorylation of mutant huntingtin might slow the progression of Huntington's disease (HD). This study showed that intraventricular infusion of GM1 ganglioside into a mouse model of HD (YAC128 mice) caused phosphorylation of mutant huntingtin at specific amino acid residues that are known to attenuate huntingtin toxicity. Moreover, GM1 treatment also reduced neuronal dysfunction and improved motor function in mice that previously showed symptoms of HD. This study highlights that GM1 — which has already been investigated in patients with other disorders — might be beneficial in HD.
ORIGINAL RESEARCH PAPER
Di Pardo, A. et al. Ganglioside GM1 induces phosphorylation of mutant huntingtin and restores normal motor behavior in Huntington disease mice. Proc. Natl Acad. Sci. USA 109, 3528–3533 (2012)Article
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GM1 ganglioside improves motor function. Nat Rev Drug Discov 11, 274 (2012). https://doi.org/10.1038/nrd3709
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DOI: https://doi.org/10.1038/nrd3709
