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Author

M. Raspa

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Open access Jul 2026

Gene supplementation therapy restores motor deficits in Gnao1[C215Y] mice and proves broad efficacy in C. elegans model.

It is demonstrated that overexpression of the wild-type protein mitigates behavioral abnormalities and restores functional neuronal deficits in young adult mice, thereby supporting the development of AAV-mediated gene augmentation strategies to counteract the effects of GNAO1 variants in patients.

Germana Cocozza, T. D’Andrea, M. Di Rocco et al. · 0 citations

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