Liver repopulation with hepatocyte transplantation: new avenues for gene and cell therapy
✍ Scribed by Sanjeev Gupta; Harmeet Malhi; S. Gagandeep; Phyllis Novikoff
- Publisher
- John Wiley and Sons
- Year
- 1999
- Tongue
- English
- Weight
- 297 KB
- Volume
- 1
- Category
- Article
- ISSN
- 1099-498X
No coin nor oath required. For personal study only.
✦ Synopsis
Liver-directed gene therapy is appropriate for many conditions. Recent work established that liver repopulation with transplanted cells can be effective in treating genetic disorders. Although hepatocytes express therapeutic genes with considerable ef®ciency, correction of genetic disorders is constrained by limitations in permanent gene transfer into hepatocytes and repopulation of the liver with transplanted cells. Adenoviral vectors are highly ef®cient for hepatic gene transfer but the onset of deleterious host immune responses against adenoviral vectors, along with clearance of transduced hepatocytes have caused problems. Nonetheless, recent work concerning engraftment and proliferation of transplanted hepatocytes in the liver has provided signi®cant new information, which should refocus interest in hepatocyte-based therapies. Moreover, hepatocyte transplantation systems offer creative tools for de®ning critical mechanisms in gene regulation and survival of transduced cells.