GM2 ganglioside

1. Cinnamic acid decreases GM2 ganglioside in mice.

   “reduced the accumulation of GM2 gangliosides/glycoconjugates in the cerebral cortex of Sandhoff mice”

   Animalsin Sandhoff micecerebral cortex

   Cinnamic acid, a natural plant compound, exhibits neuroprotection in a mouse model of Sandhoff disease via PPARα

   NeuroImmune pharmacology and therapeutics·15 Mar 2024

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1. AAV9 prevents GM2 ganglioside in mice.

   “AAV9 treatment was sufficient to prevent GM2 and GA2 storage almost completely in the cerebrum (less so in the cerebellum)”

   Animalsin Hexb-/- Sandhoff disease mice

   Intravenous administration of scAAV9-Hexb normalizes lifespan and prevents pathology in Sandhoff disease mice

   Human molecular genetics·15 Mar 2018

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