BS-514-01 SCN10ASHORT AS A NOVEL GENE THERAPY TARGET TO TREAT CONDUCTION DEFECTS

Abstract

Voltage-gated sodium channels play a critical role in the action potential (AP) upstroke velocity and impulse propagation in the heart. Sodium channel gene therapy is challenged by the relatively large transgene size of Sodium Voltage-Gated Channel Alpha Subunit 5 (SCN5A), the main alpha-subunit of the cardiac sodium channel isoform. Our previous work demonstrated that SCN10Ashort, a small fragment comprising of the C-terminus of SCN10A, increased sodium current when co-expressed with SCN5A. In the present study we therefore explored SCN10Ashort as a novel gene therapy target.