Gene therapy vectors based on adeno-associated virus: Characteristics and applications to acquired and inherited diseases (Review)

Adeno-associated virus (AAV), a defective parvovirus, was discovered more t han 30 years ago. Interest in this virus for human gene therapy application s focuses on its non-pathogenicity, broad tropism and infectivity, site-spe cific integration and long-term persistence. The field of rAAV research has considerably advanced: titers of 10(14) p/ml have been achieved, plasmid s ystems devised to produce helper-free viruses, chimaeric vectors combining properties of rAAV ITRs and large sequence capacity from Ad/HS vectors in p arallel with the revolutionary intron strategy based on heterodimerisation of the forming concatamers have expanded the vector capacity. Muscle cells and neurons (post-mitotic cells) are amongst the most efficient targets of rAAV delivery and AAV receptors and co-receptors have been identified. This review will describe advances in the field of rAAV technology that overcom e certain limitations of the vector as a gene delivery system and overview applications involving these recombinant vectors for the treatment of acqui red and inherited diseases.