Liver-directed gene transfer and application to therapy.
The liver is an important and attractive target for the development of gene therapy strategies. Many genetic diseases are manifested in the liver, and both infectious and malignant diseases affect this organ. Retroviral and adenoviral vectors have been shown to infect hepatocytes with varying efficiently in vitro and in vivo. The presence of unique receptors at the cellular membrane of hepatocytes has stimulated the development of transfer strategies based on receptor targeting of vectors. The results of a first clinical trial for gene therapy in the liver based on ex vivo gene delivery has shown both the feasibility and the limits of current technology. This review discusses both existing vectors and strategies and prospective developments towards liver-directed gene therapy of genetic and malignant diseases.
Sandig
V
V
Max Delbrück Center for Molecular Medicine, Berlin-Buch, Germany.
Strauss
M
M
D000818:Animals; D002986:Clinical Trials as Topic; D004333:Drug Administration Routes; D018014:Gene Transfer Techniques; D030342:Genetic Diseases, Inborn / Q000628:therapy*; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D006938:Hyperlipoproteinemia Type II / Q000628:therapy; D008107:Liver Diseases / Q000628:therapy*; D008113:Liver Neoplasms / Q000628:therapy; D008659:Metabolic Diseases / Q000628:therapy*