Beyond traditional liver transplantation, emerging genetic therapies are revolutionizing the treatment landscape for hereditary liver diseases. Advances in gene editing technologies, such as CRISPR/Cas9, and novel delivery systems like adeno-associated viral vectors, are enabling precise correction of disease-causing mutations at the molecular level. These approaches show promise in monogenic liver disorders such as Wilson disease, alpha-1 antitrypsin deficiency, and progressive familial intrahepatic cholestasis.
Early-phase clinical trials are demonstrating encouraging safety and efficacy profiles, suggesting a potential paradigm shift toward curative, minimally invasive treatments that may ultimately reduce or eliminate the need for transplantation. Gain in-depth insights into emerging genetic therapies for hereditary liver diseases from renowned pediatric transplant hepatologist Dr.…
