Hepatocyte transplantation for inherited metabolic diseases of the liver
Article first published online: 20 AUG 2012
© 2012 The Association for the Publication of the Journal of Internal Medicine
Journal of Internal Medicine
Volume 272, Issue 3, pages 201–223, September 2012
How to Cite
Hepatocyte transplantation for inherited metabolic diseases of the liver (Review). J Intern Med 2012; 272: 201–223., , , , , , (Karolinska University Hospital Huddinge, Stockholm, Sweden.
- Issue published online: 23 AUG 2012
- Article first published online: 20 AUG 2012
- Accepted manuscript online: 12 JUL 2012 11:25AM EST
- The Research Strategy Committee Karolinska Institute
- The Stockholm County Council
- transplantation immunology;
- α-1-antitrypsin deficiency
Inherited metabolic diseases of the liver are characterized by deficiency of a hepatic enzyme or protein often resulting in life-threatening disease. The remaining liver function is usually normal. For most patients, treatment consists of supportive therapy, and the only curative option is liver transplantation. Hepatocyte transplantation is a promising therapy for patients with inherited metabolic liver diseases, which offers a less invasive and fully reversible approach. Procedure-related complications are rare. Here, we review the experience of hepatocyte transplantation for metabolic liver diseases and discuss the major obstacles that need to be overcome to establish hepatocyte transplantation as a reliable treatment option in the clinic.