Parkinson’s disease recovery by GM1 oligosaccharide treatment in the B4galnt1 +/− mouse model

Elena Chiricozzi, Laura Mauri, Giulia Lunghi, Erika Di Biase, Maria Fazzari, Margherita Maggioni, Manuela Valsecchi, Simona Prioni, Nicoletta Loberto, Diego Yuri Pomè, Maria Grazia Ciampa, Pamela Fato, Gianluca Verlengia, Stefano Cattaneo, Robert Assini, Gusheng Wu, Samar Alselehdar, Robert W. Ledeen, Sandro Sonnino

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12 Scopus citations


Given the recent in vitro discovery that the free soluble oligosaccharide of GM1 is the bioactive portion of GM1 for neurotrophic functions, we investigated its therapeutic potential in the B4galnt1+/− mice, a model of sporadic Parkinson’s disease. We found that the GM1 oligosaccharide, systemically administered, reaches the brain and completely rescues the physical symptoms, reduces the abnormal nigral α-synuclein content, restores nigral tyrosine hydroxylase expression and striatal neurotransmitter levels, overlapping the wild-type condition. Thus, this study supports the idea that the Parkinson’s phenotype expressed by the B4galnt1+/− mice is due to a reduced level of neuronal ganglioside content and lack of interactions between the oligosaccharide portion of GM1 with specific membrane proteins. It also points to the therapeutic potential of the GM1 oligosaccharide for treatment of sporadic Parkinson’s disease.

Original languageEnglish (US)
Article number19330
JournalScientific reports
Issue number1
StatePublished - Dec 1 2019

ASJC Scopus subject areas

  • General


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