ABSTRACT
This chapter reviews the development and current applications of somatic gene therapy to the central nervous system (CNS), with particular emphasis on the ex vivo strategy. It describes the rationales for selecting a gene delivery method, and the advantages and disadvantages of different target cells for genetic manipulation. The chapter presents the examples of work to date that have applied the somatic gene therapy strategy to animal models of neurological disease. In designing gene therapy for neurologic diseases in the brain, it may be important to deliver genes focally in the adult brain. Somatic gene therapy, or the genetic manipulation of nongerm line cells for therapeutic applications, has emerged as one of the most promising strategies for treating human disease. The chapter concludes with a note on some of the future directions of somatic gene therapy for the CNS.
