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Hepatocyte transplantation for inherited metabolic diseases of the liver

  1. C. Jorns1,*,
  2. E. C. Ellis1,
  3. G. Nowak1,
  4. B. Fischler2,
  5. A. Nemeth2,
  6. S. C. Strom3,
  7. B. G. Ericzon1

Article first published online: 20 AUG 2012

DOI: 10.1111/j.1365-2796.2012.02574.x

Issue

Journal of Internal Medicine

Journal of Internal Medicine

Volume 272, Issue 3, pages 201–223, September 2012

Additional Information

How to Cite

Jorns C, Ellis EC, Nowak G, Fischler B, Nemeth A, Strom SC, Ericzon BG (Karolinska University Hospital Huddinge, Stockholm, Sweden. Hepatocyte transplantation for inherited metabolic diseases of the liver (Review). J Intern Med 2012; 272: 201–223.

Author Information

  1. 1

    Division of Transplantation Surgery, Department of Clinical Science, Intervention and Technology (CLINTEC), Karolinska University Hospital Huddinge, Stockholm, Sweden

  2. 2

    Division of Pediatrics, Department of Clinical Science, Intervention and Technology (CLINTEC), Karolinska University Hospital Huddinge, Stockholm, Sweden

  3. 3

    Division of Pathology, Department of Laboratory Medicine Karolinska Institutet, Karolinska University Hospital Huddinge, Stockholm, Sweden

*Correspondence: Carl Jorns MD, Division of Transplantation Surgery, Department of Clinical Science, Intervention and Technology (CLINTEC), Karolinska Institute, Karolinska University Hospital Huddinge, Stockholm, 14186, Sweden. (fax: +4687743191; e-mail: carl.jorns@ki.se).

Publication History

  1. Issue published online: 23 AUG 2012
  2. Article first published online: 20 AUG 2012
  3. Accepted manuscript online: 12 JUL 2012 11:25AM EST

Funded by

  • The Research Strategy Committee Karolinska Institute
  • The Stockholm County Council

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Keywords:

  • experimental;
  • hepatology;
  • liver;
  • surgery;
  • transplantation immunology;
  • α-1-antitrypsin deficiency

Abstract

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.

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