Abstract:Autism spectrum disorder (ASD) is a highly heritable neurodevelopmental disorder with deficits in social communication and stereotypical behaviors. Whole-brain genome editing to correct single-base mutations and alleviate autistic-like behaviors in animal models has not been achieved. Here we developed an APOBEC-embedded cytosine base editor (AeCBE) system, for converting C·G to T·A base pairs. We demonstrate the effectiveness by targeting AeCBE to an ASD-associated mutation of the MEF2C gene (c.104T>C, p.L… Show more
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