Key result
A novel de novo KCNA1(I262M) mutation causing episodic ataxia type I leads to a defective voltage-gated potassium channel with a dominant-negative effect in vitro.
Case Report
The novel KCNA1(I262M) mutation causes episodic ataxia type I through a dominant-negative effect on voltage-gated potassium channel function.
No immediate clinical change; leaves open whether this KCNA1 variant informs targeted therapies in episodic ataxia.
We describe the clinical phenotype of a novel de novo KNCA1 mutation, and functional characterization of the effects of the mutation on Kv1.1 channel function. HEK293 cells were transfected transiently with either wild-type or mutant channels. Representative currents were evoked after application of a series of square voltage steps from -80 mV to +50 mV in 200-ms intervals from Vh = -80 mV. Extracellular K(+) was added to evoke tail currents. Equal amounts of wild-type and Kv1.1(I262M) mutant DNA were transfected transiently in HEK293 cells to evaluate the influence of the mutation. We found that Kv1.1(I262M) leads to a defective voltage-gated potassium channel. Coexpression studies revealed a dominant-negative effect. We describe the phenotype of a novel KCNA1 mutation causing episodic ataxia. Patch-clamp studies confirm the pathogenicity of the mutation in vitro and suggest that it is dominant with respect to wild-type.
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Lassche et al. (2014) conducted a case report in Episodic ataxia type I. KCNA1(I262M) mutation vs. Wild-type Kv1.1 channels was evaluated on Kv1.1 channel function. A novel de novo KCNA1(I262M) mutation causing episodic ataxia type I leads to a defective voltage-gated potassium channel with a dominant-negative effect in vitro.
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