Transferência de gene como tratamento

Abstract

Objective

To study gene transfer aiming at its future clinical applications in the treatment of inborn metabolic diseases of the liver.

Methods

Review of the literature.

Results and Conclusions

Gene transfer into the liver can be an alternative to liver transplantation in the treatment of inborn metabolic diseases. Various vectors have been described in gene transfer, including retrovirus vectors, whose integration into the chromosomal DNA can allow stable, long-term expression of the transgene. The integration of retrovirus vectors into the genome of the target cell is possible only during mitosis. Therefore, these vectors must be delivered during hepatic regeneration induced by partial hepatectomy, for example. Another obstacle that needs to be overcome is avoiding extrahepatic dissemination of retroviruses, especially to germinal cells, since that would present the risk of changing the genetic heritage of descendants.