Use of Recombinant Adenovirus for Gene Transfer into the Rat Brain: Evaluation of Gene Transfer Efficiency, Toxicity, and Inflammatory and Immune Reac

Adenovirus (Ad) vectors have been used to successfully deliver genes into a wide variety of non-central nervous system (CNS) tissues and organs in animal models of human disease and in several human phase I clinical trials (1 –3 ). Adenoviruses are easily purified to the high titers required for in vivo administration and they are efficient in transducing terminally differentiated cells such as neurons and glial cells, resulting in high levels of transgene expression and spatially restricted transgene expression within the region of virus administration. To date, most vectors utilized have been first-generation partially deleted vectors. The most commonly used have been E1/E3-deleted vectors, or second- and third-generation vectors with further deletions in E2 orE4.