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Hepatocyte transplantation

Namita Roy-Chowdhury, PhD
Jayanta Roy-Chowdhury, MD, MRCP
Section Editor
Robert S Brown, Jr, MD, MPH
Deputy Editor
Kristen M Robson, MD, MBA, FACG


Advances in the understanding of hepatocyte engraftment and the remarkable proliferative potential of hepatocytes have brought liver cell transplantation to the doorstep of application in the treatment of inherited and acquired human diseases. Extensive animal experiments have shown that hepatocytes transplanted in the liver or at ectopic sites survive, function, and participate in the regenerative process. Because the host liver architecture remains intact following the integration of the engrafted hepatocytes in the liver cords, hepatocyte transplantation is metabolically less stressful than transplantation of the whole organ, and the consequences of graft loss are much less severe.

Hepatocyte transplantation has many potential applications. Therapeutic genes can be transferred into cultured hepatocytes, and the phenotypically modified cells can then be transplanted for ex vivo gene therapy. Such gene transfer could be used to replace a missing gene product, to prevent immune rejection, or to give the cells a proliferative advantage. Hepatocyte transplantation does not interfere with subsequent liver transplantation or gene therapy. Although the clinical efficacy of hepatocyte transplantation has been demonstrated, the shortage of good quality donor livers for hepatocyte isolation and the lack of dependable methods of cryopreservation will limit widespread clinical application of this method until further research overcomes these problems.


Potential clinical applications of hepatocyte transplantation are listed in (table 1).

Treatment of inherited metabolic diseases — Missing gene products can be substituted by transplanting normal primary hepatocytes from allogeneic donors. This simple approach holds promise for diseases such as Crigler-Najjar syndrome type 1, urea cycle disorders, and coagulopathies, including hemophilias. (See "Crigler-Najjar syndrome" and "Inborn errors of metabolism: Classification", section on 'Urea cycle disorders' and "Genetics of the hemophilias".)

Hepatocytes used for gene therapy can be derived from a separate donor (allogeneic transplantation) or the recipient can serve as the donor, in which case the genetic defect needs to be corrected before transplantation (autologous transplantation). An advantage of autologous transplantation is that it does not require immunosuppression [1-3].

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Literature review current through: Nov 2017. | This topic last updated: Jul 25, 2017.
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