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Aicardi-Goutieres Syndrome Associated ADAR G1007R mutation dominantly induces neuroinflammation in mouse brain

Guo, X.; Liu, J.-J.; Shang, C.; Guerriero, C.; Wiley, C.; Steinman, R.; Sheng, Y.; Brodsky, J.; Billiar, T.; Liu, S.; Wang, Q.

2025-04-21 neuroscience
10.1101/2025.04.21.649787 bioRxiv
Show abstract

The ADARG1007Rmutation is one of the most frequent mutations found in type six Aicardi-Goutieres Syndrome (AGS), a severe inflammatory encephalopathy in pediatric patients. We report here a mouse model bearing an human equivalent ADARG1007R mutation, and the heterozygous mice recapitulated some pathologic features of ADARG1007R AGS patients, including early-onset brain inflammation in heterozygous individuals and interferon-stimulated gene (ISG) expression within deep brain areas. Furthermore, we demonstrated that brain inflammation could be reversed by deletion of the cellular RNA receptor MDA5, which blocks the cellular RNA sensing signaling pathway. This model provides a unique tool for studying the molecular mechanisms underlying the heterozygous ADARWT/G1007R mutation in AGS brain pathogenesis. It may also be a valuable platform for developing personalized therapies for patients with this specific mutation.

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