Single Construct Suppression and Replacement Gene Therapy for the Treatment of All CALM1 -, CALM2 -, and CALM3 -Mediated Arrhythmia Disorders.
Samantha K HamrickC S John KimDavid J TesterManuela GencarelliKathryn E TobertMartina GluscevicMichael J AckermanPublished in: Circulation. Arrhythmia and electrophysiology (2024)
CaM-mediated long-QT syndrome induced pluripotent stem cell-derived CM lines. The single CALM-SupRep construct may be able to treat all calmodulinopathies, regardless of which of the 3 CaM-encoding genes are affected.