A heterozygous deletion mutation in the cardiac sodium channel gene SCN5A with loss- and gain-of-function characteristics manifests as isolated conduction disease, without signs of Brugada or long QT syndrome

PLoS One. 2013 Jun 28;8(6):e67963. doi: 10.1371/journal.pone.0067963. Print 2013.

Abstract

Background: The SCN5A gene encodes for the α-subunit of the cardiac sodium channel NaV1.5, which is responsible for the rapid upstroke of the cardiac action potential. Mutations in this gene may lead to multiple life-threatening disorders of cardiac rhythm or are linked to structural cardiac defects. Here, we characterized a large family with a mutation in SCN5A presenting with an atrioventricular conduction disease and absence of Brugada syndrome.

Method and results: In a large family with a high incidence of sudden cardiac deaths, a heterozygous SCN5A mutation (p.1493delK) with an autosomal dominant inheritance has been identified. Mutation carriers were devoid of any cardiac structural changes. Typical ECG findings were an increased P-wave duration, an AV-block I° and a prolonged QRS duration with an intraventricular conduction delay and no signs for Brugada syndrome. HEK293 cells transfected with 1493delK showed strongly (5-fold) reduced Na(+) currents with altered inactivation kinetics compared to wild-type channels. Immunocytochemical staining demonstrated strongly decreased expression of SCN5A 1493delK in the sarcolemma consistent with an intracellular trafficking defect and thereby a loss-of-function. In addition, SCN5A 1493delK channels that reached cell membrane showed gain-of-function aspects (slowing of the fast inactivation, reduction in the relative fraction of channels that fast inactivate, hastening of the recovery from inactivation).

Conclusion: In a large family, congregation of a heterozygous SCN5A gene mutation (p.1493delK) predisposes for conduction slowing without evidence for Brugada syndrome due to a predominantly trafficking defect that reduces Na(+) current and depolarization force.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Action Potentials / genetics
  • Arrhythmias, Cardiac / genetics*
  • Arrhythmias, Cardiac / metabolism
  • Arrhythmias, Cardiac / pathology
  • Brugada Syndrome / genetics*
  • Brugada Syndrome / metabolism
  • Brugada Syndrome / pathology
  • Cardiac Conduction System Disease
  • Cell Line
  • Death, Sudden, Cardiac / pathology
  • Electrocardiography / methods
  • Female
  • HEK293 Cells
  • Heart / physiopathology
  • Heart Conduction System / abnormalities*
  • Heart Conduction System / metabolism
  • Heart Conduction System / pathology
  • Heterozygote
  • Humans
  • Long QT Syndrome / genetics*
  • Long QT Syndrome / metabolism
  • Long QT Syndrome / pathology
  • Male
  • Middle Aged
  • NAV1.5 Voltage-Gated Sodium Channel / genetics*
  • NAV1.5 Voltage-Gated Sodium Channel / metabolism
  • Pedigree
  • Sarcolemma / genetics
  • Sarcolemma / metabolism
  • Sarcolemma / pathology
  • Sequence Deletion / genetics*
  • Sodium / metabolism
  • Sodium Channels / genetics*
  • Sodium Channels / metabolism

Substances

  • NAV1.5 Voltage-Gated Sodium Channel
  • SCN5A protein, human
  • Sodium Channels
  • Sodium

Grants and funding

This work was supported by grants of the Fondation Leducq, Paris, France, the German Research Foundation, Bonn, Germany (DFG; SFB 656-C1) and the Interdisciplinary Center for Clinical Research, Münster, Germany (IZKF; Schu01-012-11) to ES-B as well as by grants of the Innovative medicine research, Münster, Germany (IMF; ST121119) to BS. The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.