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Novel CALM3 Variant Causing Calmodulinopathy With Variable Expressivity in a 4-Generation Family
Journal article   Open access   Peer reviewed

Novel CALM3 Variant Causing Calmodulinopathy With Variable Expressivity in a 4-Generation Family

Koichi Kato, Holly M Isbell, Véronique Fressart, Isabelle Denjoy, Amal Debbiche, Hideki Itoh, Jacques Poinsot, Alfred L George Jr, Alain Coulombe, Madeline A Shea, …
Circulation. Arrhythmia and electrophysiology, Vol.15(3), e010572
02/28/2022
DOI: 10.1161/CIRCEP.121.010572
PMID: 35225649
url
https://doi.org/10.1161/CIRCEP.121.010572View
Published (Version of record) Open Access

Abstract

CaM (calmodulin), encoded by 3 separate genes ( , , and ), is a multifunctional Ca -binding protein involved in many signal transduction events including ion channel regulation. CaM variants may present with early-onset long QT syndrome (LQTS), catecholaminergic polymorphic ventricular tachycardia, or sudden cardiac death. Most reported variants occurred de novo. We identified a novel variant, p.Asn138Lys (N138K), in a 4-generation family segregating with LQTS. The aim of this study was to elucidate its pathogenicity and to compare it with that of p.D130G-CaM-a variant associated with a severe LQTS phenotype. We performed whole exome sequencing for a large, 4-generation family affected by LQTS. To assess the effect of the detected variant, the intrinsic Ca -binding affinity was measured by stoichiometric Ca titrations and equilibrium titrations. L-type Ca and slow delayed rectifier potassium currents (I and I ) were recorded by whole-cell patch-clamp. Cav1.2 and Kv7.1 membrane expression were determined by optical fluorescence assays. We identified 14 p.N138K-CaM carriers in a family where 2 sudden deaths occurred in children. Several members were only mildly affected compared with CaM-LQTS patients to date described in literature. The intrinsic Ca -binding affinity of the CaM C-terminal domain was 10-fold lower for p.N138K-CaM compared with WT-CaM. I inactivation was slowed in cells expressing p.N138K-CaM but less than in p.D130G-CaM cells. Unexpectedly, a larger I current density was observed in cells expressing p.N138K-CaM, but not for p.D130G-CaM, compared with WT-CaM. The p.N138K variant impairs Ca -binding affinity of CaM and I inactivation but potentiates I . The variably expressed phenotype of this variant compared with previously published de novo LQTS-CaM variants is likely explained by a milder impairment of I inactivation combined with I augmentation.
Ion Channels Phenotype potassium long QT syndrome calmodulin

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