In vivo hepatic gene therapy
Journal
Acta Paediatrica Taiwanica
Journal Volume
42
Journal Issue
4
Pages
191-200
Date Issued
2001
Author(s)
Abstract
Gene therapy is intended to treat diseases at the gene level by replacing a missense gene with a normal gene or repairing a mutated gene and letting right gene function normally. Hepatic gene therapy applies this idea to the areas of inherited and metabolic liver disease, liver cancer, viral hepatitis, or even the systemic diseases, like hemophilia A and B. The strategies of hepatic gene therapy can be divided into two categories: ex vivo and in vivo. The ex vivo method has to harvest the hepatocytes from the hosts and introduce the gene of interest into the hepatocytes and retransplant the cells back to the liver. The in vivo method constitutes either a local delivery method or a systemic administration. The vectors for in vivo gene transfer include viral or non-viral methods. For the viral methods, retrovirus, lentivirus, adenovirus, adeno-associated virus, or baculovirus had been tried. For the non-viral methods, liposome, liver-specific ligand, or even naked nucleotide had been attempted to achieve the goal of liver-directed gene transfer. Up to now, neither viral nor non-viral vector is perfect. A further modification of the current vectors may improve a new generation of liver-directed gene transfer.
Subjects
Asialoglycop-rotein; Hepatic gene therapy; Liposome; Non-viral vector; Viral vector
SDGs
Other Subjects
ligand; liposome; nucleotide derivative; Adeno associated virus; Adenovirus; Baculovirus; expression vector; gene replication; gene therapy; gene transfer; genetic disorder; hemophilia A; hemophilia B; human; Lentivirinae; liver cancer; liver disease; metabolic disorder; missense mutation; Retrovirus; review; virus hepatitis; Adenoviridae; Clinical Trials; Gene Therapy; Gene Transfer Techniques; Genetic Vectors; Humans; Liposomes; Liver Diseases; Prognosis; Retroviridae; Sensitivity and Specificity
Type
review