Table 1Some liver-based disorders that are current targets for gene therapy

I. Inherited Liver-Based Disorders
• Disorders causing damage to hepatocytes or to the liver architecture:
α1-antitrypsin deficiency
Glycogen storage diseases, e.g., von Gierke's disease and Pompe's disease
Wilson's disease
Progressive familial intrahepatic cholestasis
Tyrosinemia
• Disorders that do not affect hepatocyte longevity or liver architecture:
Crigler-Najjar syndrome type I
Familial hypercholesterolemia and other lipid metabolic disorders
Mucopolysaccharidosis VII
Ornithine transcarbamylase deficiency
Maple syrup urine disease
Phenylketonuria
Hemophilia A and B
Oxalosis
II. Acquired Diseases
• Infectious diseases:
Hepatitis B and C (prophylaxis and treatment), and malaria (prophylaxis).
• Liver tumors:
Hepatomas, cholangiocarcinomas, metastatic tumors
Extrahepatic tumors (inhibition of neovascularization)
• Cirrhosis of the liver
• Allograft or xenograft rejection

From: Hepatocyte Transplantation and Liver-Directed Gene Therapy

Cover of Madame Curie Bioscience Database
Madame Curie Bioscience Database [Internet].
Austin (TX): Landes Bioscience; 2000-2013.
Copyright © 2000-2013, Landes Bioscience.

NCBI Bookshelf. A service of the National Library of Medicine, National Institutes of Health.