Current development of adeno-associated viral vectors.
Vectors based on adeno-associated virus (AAV) have recently been used in phase I clinical trials for the treatment of neurological disorders, such as Parkinson's and Canavan's diseases. Indeed, AAV-mediated gene transfer is a promising tool for the delivery of therapeutic gene into the central and peripheral nervous systems. AAV-mediated gene transfer was also applied in phase I and phase II clinical trials for the treatment of cystic fibrosis and in phase I clinical trials for the treatment of hemophilia B. Remarkable progress is being reported in the development of AAV-based vectors; however, the design of AAV-derived vectors needs to be improved. As it stands, AAV-mediated gene transfer has a limited capacity in accommodating foreign genes. In addition, some preclinical studies have shown that AAV-derived vectors can cause tumors in animals due to insertional mutagenesis events. This review will discuss perspectives and drawbacks for AAV-based vector systems.
Romano
G
G
Department of Neurosurgery, The Farber Institute for the Neurosciences, Jefferson Medical College, Thomas Jefferson University, Philadelphia, PA 19107, USA. Gaetano.Romano@jefferson.edu
D000256:Adenoviridae / Q000235:genetics*; D000818:Animals; D017825:Canavan Disease / Q000235:genetics / Q000628:therapy; D002986:Clinical Trials as Topic; D003550:Cystic Fibrosis / Q000235:genetics / Q000628:therapy; D000229:Dependovirus / Q000235:genetics*; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D009154:Mutation; D010300:Parkinson Disease / Q000235:genetics / Q000628:therapy