Article

Gene therapy for targeting a prenatally enriched potassium channel associated with severe childhood epilepsy and premature death

2024-10-24

Abstract excerpt

Dysfunction of the sodium-activated potassium channel KNa1.1 (encoded by KCNT1) is associated with a severe condition characterized by frequent seizures (up to hundreds per day) and is often fatal by age three years. We defined the early developmental onset of KNa1.1 channels in prenatal and neonatal brain tissue, establishing a timeline for pathophysiology and a window for therapeutic intervention. Using patch-cl...

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Identifiers and source

Literature Corpus work: b7760632-547b-5153-9eff-9da678f259a8
DOI: 10.1101/2024.10.24.620125

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