Article

Rett syndrome lifespan extension in mice via AI-guided ADAR editing

2026-06-28

Abstract excerpt

Rett syndrome is a severe neurodevelopmental disorder primarily caused by mutations in the MECP2 gene. A significant subset of severe cases are driven by nonsense mutations that generate premature stop codons, leading to loss of functional MeCP2 protein. Here, we describe a novel therapeutic strategy that uses endogenous adenosine deaminase acting on RNA (ADAR) enzymes to correct the R168X mutation at the RNA lev...

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Literature Corpus work: 2814fe1e-4684-55ff-a831-c33538d54288
DOI: 10.64898/2026.06.23.734060

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