In vivo base editing alleviates hepatic iron accumulation and fibrosis in models of HFE-related hereditary hemochromatosis

The authors investigate the efficacy and safety of in vivo base editing to correct the HFE C282Y mutation responsible for hereditary hemochromatosis, which leads to excessive iron accumulation and liver damage. Using lipid nanoparticles to deliver the editing components, they demonstrate significant reductions in hepatic iron levels and associated fibrosis in murine models and patient-derived cells, with no off-target effects detected. This study provides a proof of concept for a potential therapeutic approach to address HFE-related iron overload disorders.