Liver: A Major Target Organ in Gene Therapy

The liver is the largest and most blood-rich solid organ in the body, with multiple important biological functions essential for maintaining good health of animals and humans. The liver consist of hepatocytes (60–65% of total liver cells), sinusoidal endothelial cells (15–20%), Kupffer cells (8–12%), hepatic stellate cells (HSCs) or Ito cells (3–8%), biliary epithelial cells (3–5%), liver dendritic cells (<1%), cells constituting blood vessels, and blood cell components passing through the hepatic circulation. Hepatocytes produce, secrete, store, and/or degrade various molecules essential for normal biological activities of organisms. Hepatic dendritic cells (DCs), Kupffer cells (KCs), sinusoidal endothelial cells, and hepatocytes in some cases, serve as antigen presenting cells (APCs). Disruption of these biological functions and metabolic homeostasis in the liver