Gene therapy using AAV vectors

Abstract

AAV (adeno-associated virus) vectors are considered to be promising gene-delivery vehicles for gene therapy, because they are derived from non-pathogenic virus, efficiently transduce non-dividing cells, and cause long-term gene expression. Appropriate AAV serotypes are utilized depending on the type of target cells. Among various neurological disorders, Parkinson's disease is one of the most promising candidates of gene therapy. On the other hand, intramuscular injection of AAV vectors is appropriate to protein-supplement gene therapy. Monogenic diseases such as hemophilia are suitable candidates. © 2007, THE JAPAN SOCIETY OF DRUG DELIVERY SYSTEM. All rights reserved.