Rhes depletion and mutant huntingtin accumulation in HD mice!

Published on August 10, 2023

Imagine the striatum (a brain region) as a bustling city. The mayor of this city is called Rhes, who has an important job in maintaining order and balance. But in Huntington’s disease (HD), trouble arises when mutant huntingtin (a harmful protein) starts piling up in the city. Our study shows that when we knock down Rhes, the mutant huntingtin levels go through the roof, causing damage to the neurons and increasing astrogliosis (the condition where support cells in the brain become reactive). However, this only happens during the early stages of HD, not when symptoms are already present. It turns out that Rhes depletion affects the autophagy process, which is like the city’s waste management system. Autophagy clears out unwanted proteins, but when Rhes is lacking, this system doesn’t work properly, leading to the accumulation of mutant huntingtin in the striatum. This discovery sheds light on how Rhes functions in HD and opens new doors for potential treatments. If you want to learn more about our exciting research, click the link below!

IntroductionHuntington’s disease (HD) is caused by CAG trinucleotide repeats in the HTT gene. Selective neurodegeneration in the striatum is prominent in HD, despite widespread expression of mutant HTT (mHTT). Ras homolog enriched in the striatum (Rhes) is a GTP-binding protein enriched in the striatum, involved in dopamine-related behaviors and autophagy regulation. Growing evidence suggests Rhes plays a critical role in the selective striatal degeneration in HD, but its specific function in this context remains complex and controversial.MethodsIn this study, we utilized CRISPR/Cas9 to knockdown Rhes at different disease stages through adeno-associated virus (AAV) transduction in HD knock-in (KI) mice. Immunoblotting and immunofluorescence were employed to assess the impact of Rhes depletion on mHTT levels, neuronal loss, astrogliosis and autophagy activity.ResultsRhes depletion in 22-week-old HD KI mice (representing the presymptomatic stage) led to mHTT accumulation, reduced neuronal cell staining, and increased astrogliosis. However, no such effects were observed in 36-week-old HD KI mice (representing the symptomatic stage). Additionally, Rhes deletion in 22-week-old HD KI mice resulted in increased P62 levels, reduced LC3-II levels, and unchanged phosphorylation of mTOR and beclin-1, unchanged mTOR protein level, except for a decrease in beclin-1.DiscussionOur findings suggest that knockdown Rhes promotes striatal aggregation of mutant huntingtin by reducing autophagy activity in a mTOR-independent manner. Rhes plays a protective role during the presymptomatic stage of HD KI mice.

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