Gene therapy for treatment of cerebral ischemia using defective recombinant adeno-associated virus vectors.

In this review we present our results and experiences in performing gene therapy of cerebral stroke using recombinant adeno-associated virus (rAAV) vectors in a rat model. The methodologies involving the production of AAV vectors, gene transfer to the brain, and a trivessel ligation model of focal ischemic cerebral stroke in rats are described. Furthermore, a brief description of other viral vectors and candidates of therapeutic transgenes used for gene therapy of cerebral stroke are presented. The potential advantages and limitations of stroke gene therapy are also discussed with the intention of outlining the design of more appropriate experiments. Copyright 2002 Elsevier Science (USA)