Ellie M. Chilcott

This study compares AAV9 and AAV-F capsids for treating PDHD in mice, finding AAV-F improves survival and enzyme activity at lower doses but causes motor impairments.

This paper demonstrates the use of AAV9 vectors to deliver AntagoNATs targeting a natural antisense transcript in a DS mouse model, improving survival and reducing seizures.

This study demonstrates that AAV-delivered RNA editing machinery improves survival and reduces seizures in a mouse model of Dravet Syndrome.

This study investigates spinal cord pathology in a Dravet Syndrome mouse model and identifies dysregulated pathways related to monooxygenase activity, fatty acid ligases, and lactate transporters.