GENE-TRANSFER BY ADENOASSOCIATED VIRUS VECTORS INTO THE CENTRAL-NERVOUS-SYSTEM

Adeno-associated virus (AAV) vectors are derived from a nonpathogenic and defective human parvovirus. Although currently unable to display t he integration specificity featured by its wild-type parent, the recom binant AAV (rAAV) system has continued to attract enormous interest pr imarily due to its unique features such as safety, high titers, broad host range, transduction of quiescent cells, and vector integration. R ecently, rAAV-mediated in vivo gene transfers have demonstrated effici ent long-term transduction (from 3 months to more than 1.5 years) and lack of cytotoxicity and cellular immune responses in the target tissu es, especially in the CNS. Alternative approaches using rAAV plasmid D NA in nonviral gene delivery systems also generated promising results. Propelled by various efforts to improve the system, rAAV vectors will provide numerous opportunities to explore the potential therapeutic a pplications in humans. (C) 1997 Academic Press.