| 抄録 |
Liver diseases, including inherited metabolic defects of genetic etiology, chronic viral hepatitis, liver cirrhosis and primary and metastatic liver cancers, represent a major health problem due to their high prevalence and limited availability of therapeutic options. Gene therapy, a procedure based on the transfer of therapeutic genes to tissue, has been considered in managing liver diseases. Initial promising results from gene therapy strategies in treating liver disease were followed by skepticism of the actual clinical value due to limitations in specificity and efficacy, and toxicity. However, new progress in demonstrating, pre-clinically, that many forms of liver diseases are amenable to treatment with gene transfer methodologies has led to clinical arena of testing genetic corrections in inherited metabolic disorders, genetic vaccines against chronic viral infection, and a diversity of gene transfer strategies for the treatment of liver cancer. These studies have shown that gene therapy is safe and can generate benefit. This presentation will summarize the results of the most recent clinical trials of gene therapy on liver diseases and new promising approaches that could fill the therapeutic need for treatment of other liver conditions. |
