title: In vivo hepatic gene therapy.
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.
authors.affiliation: Department of Pediatrics, College of Medicine, National Taiwan University Hospital, 7, Chung-Shan South Road, Taipei, Taiwan 100. yhni@ha.mc.ntu.edu.tw
authors.forename: Y H
authors.initials: YH
authors.lastname: Ni
mesh_terms: D000256:Adenoviridae / Q000235:genetics; D002986:Clinical Trials as Topic; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000009:adverse effects / Q000379:methods*; D005822:Genetic Vectors / Q000008:administration & dosage; D006801:Humans; D008081:Liposomes; D008107:Liver Diseases / Q000235:genetics / Q000628:therapy*; D011379:Prognosis; D012190:Retroviridae / Q000235:genetics; D012680:Sensitivity and Specificity
chemical_list: D008081:Liposomes