Mislocalization of KCNQ2 Channels as a Pathogenic Mechanism in KCNQ2 Developmental and Epileptic Encephalopathy.

Document Type

Article

Publication Date

1-21-2026

Keywords

JGM, Animals, KCNQ2 Potassium Channel, Mice, Female, Male, Humans, Hippocampus, Protein Transport, Mice, Inbred C57BL, Epilepsy, Mice, Transgenic, HEK293 Cells, Nerve Tissue Proteins

JAX Source

J Neurosci. 2026;46(3):e0947252025.

ISSN

1529-2401

PMID

41419331

DOI

https://doi.org/10.1523/JNEUROSCI.0947-25.2025

Abstract

KCNQ2 potassium channel variants are linked to developmental and epileptic encephalopathy (DEE). However, the mechanisms by which pathogenic variants, especially those outside known hotspots, such as the S4-S5 linker, lead to disease remain unknown. Here, we examined the H228R variant, a pathogenic mutation in the S4-S5 linker associated with DEE. We tested whether H228R induces KCNQ2 channel mistargeting in addition to its biophysical effects, given recent evidence of impaired trafficking in KCNQ2 DEE variants. We confirmed the H228R variant as a loss-of-function (LOF) when expressed as a homomer and as a dominant-negative when coexpressed with wild-type (WT) KCNQ3. Surprisingly, it exhibited some gain-of-function effects when coexpressed with WT KCNQ2. To determine its cellular localization in vivo, we used male and female heterozygous

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