Adeno-associated virus vectors: versatile tools for in vivo gene transfer.
Over the last few years, viral vectors based on the adeno-associated virus have gained increasing popularity due to several favorable characteristics, including the high efficiency of transduction of postmitotic tissues in vivo and the long-term persistence of transgene expression in the absence of inflammation or immune response. Recently, completed trials have substantially confirmed the clinical applicability of these vectors, while indicating that further developments in vector design and production scale are necessary to broaden human application. This review summarizes our current knowledge on the molecular biology of these vectors and their clinical utilization, in particular concerning their application in renal gene transfer.
Zentilin
Lorena
L
Molecular Medicine Laboratory, International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.
Giacca
Mauro
M
D000229:Dependovirus / Q000145:classification / Q000235:genetics*; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D014644:Genetic Variation; D005822:Genetic Vectors; D016679:Genome, Viral; D006801:Humans; D009369:Neoplasms / Q000235:genetics / Q000628:therapy; D009422:Nervous System Diseases / Q000235:genetics / Q000628:therapy