Nonneurotropic adenovirus: a vector for gene transfer to the brain and gene therapy of neurological disorders.

Publisher Summary This chapter reviews the vectors available for gene transfer into the brain. The chapter explores the potential application of replication deficient adenovirus vectors as vehicles for gene delivery into the central nervous system (CNS). A significant feature of adenovirus as a potential vector for DNA delivery in human gene therapy protocols is that a live (replication-competent) vaccine, to provide protection against natural adenovirus infections. Similar replication-competent adenovirus recombinants encoding defined immunogens have also been used in human vaccine trials, or as replication-competent vectors with or without additional anticancer genes to induce tumor cell killing. There are protocols and methodologies involved in constructing and utilizing adenovirus vectors for gene transfer to cells of the brain both in vitro and in vivo . The chapter focuses on progress in the application of vectors for their exploitation for neurobiology and discusses potential future developments in this field. The main advantages of adenovirus-derived vectors includes a capacity to transduce both immature neonatal and mature neurons, their use to elucidate neuronal morphology and connectivity and an ability to achieve multiple infections of a single target cell with separate vector virions.