Hepatic gene therapy using adeno-associated virus vectors.
Recombinant adeno-associated virus vectors have been shown to safely transduce a number of tissues in preclinical animal studies. The level of gene transfer is sufficient to successfully treat a large number of medical disorders. Moreover, the long-term persistence of the vector sequences in animals makes it likely that this vector will be useful for genetic diseases requiring life-long therapy. This review outlines the biological principles of the vector, as well as its advantages and current limitations as it relates to use in hepatic gene therapy.
Patijn
G A
GA
Department of General Surgery, Leiden University Medical Center, The Netherlands.
Kay
M A
MA
D000818:Animals; D000229:Dependovirus / Q000235:genetics*; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D008099:Liver / Q000378:metabolism; D008107:Liver Diseases / Q000235:genetics / Q000628:therapy*