NASH is characterized by multiple underlying genetic mutations, with no approved cure to date. Gene therapies that target those genetic mutations may play a major role in treating this disease, once delivered specifically to the hepatocytes.
K. Ebeid et al. present the synthesis and the characterization of a gene delivery system capable of targeting hepatocytes by exploiting the overexpression of asialoglycoprotein receptors on their cell surface. For the authors, this formulation appears as “a promising tool to treat any type of genetic abnormality that arises in hepatocytes, and specifically NASH.”