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Argonaute in vivo

A growing focus of our lab is studying Argonaute biology in vivo. Recent discoveries have shown that missense mutations in Argonaute genes cause Argonaute Syndrome, a rare and debilitating neurodevelopmental disorder. We aim to understand how these mutations disrupt microRNA function in the brain, and how those disruptions might be therapeutically corrected.

For patient-focused information and community resources, see the AGO Alliance website.

In parallel, we use mouse models to map miRNA-target interactions across tissues and disease states, including cancer and development, to understand how small RNA regulatory networks function in vivo.

RELEVANT PUBLICATIONS

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