In Vivo Base Editing of Scn5a Rescues Type 3 Long QT Syndrome in Mice

CONCLUSIONS: These findings show that in vivo AAV9-ABEmax editing can correct the variant Scn5a allele, effectively ameliorating arrhythmia phenotypes. Our results offer a proof of concept for the treatment of hereditary arrhythmias.PMID:37965733 | DOI:10.1161/CIRCULATIONAHA.123.065624
Source: Circulation - Category: Cardiology Authors: Source Type: research