Figure 2.

Gene therapy strategies. Gene therapy strategies to treat inherited arrhythmias can act at the level of DNA or mRNA, depending on the genetic mechanism. Abbreviations: rAAVs, recombinant adeno-associated virus; CaMKII, Ca²⁺/calmodulin-dependent protein kinase II; CPVT, catecholaminergic polymorphic ventricular tachycardia; PTC, premature termination codon; NHEJ, non-homologous end-joining; HDR, homology-directed repair; ssODN, single-stranded oligonucleotide; GoF, gain-of-function, shRNA, short hairpin RNA; AON, antisense oligonucleotides; PTM, pre-trans-splicing molecule.