Hepatocyte genomic DNA (gDNA)

Hepatocyte genomic DNA refers to the complete set of genetic instructions contained within the nucleus of a liver cell. It serves as the fundamental template for the synthesis of proteins essential for metabolism, detoxification, and blood coagulation. In modern medicine, this genomic DNA is a primary target for advanced therapeutic modalities, including CRISPR-based gene editing and Adeno-associated virus (AAV)-mediated gene therapy, which aim to permanently correct or supplement defective genes [1, 4]. For instance, therapies targeting specific loci within the hepatocyte genome can treat conditions like transthyretin amyloidosis by knocking out the TTR gene or hemophilia by introducing functional clotting factor genes [1, 4]. Additionally, the integration of viral DNA, such as that from the Hepatitis B virus, into the hepatocyte genome is a major driver of chronic liver disease and hepatocellular carcinoma [3]. While targeting the genome offers the potential for curative treatments, it presents significant challenges, including the risk of off-target mutations and the long-term consequences of permanent genetic modification [2].

Other names

Liver cell DNAHepatic nuclear DNAHepatocyte genome

In vivo gene editing, gene addition, and DNA modification.