22 Progress toward Gene Therapy for Nervous System Diseases

Abstract

Gene therapy may be defined as the introduction of functional genetic material into somatic cells for therapeutic purposes. During the last decade, we have seen an impressive development of this field, the coining of concepts, the initial application of emerging techniques, and the evolution of new tools based on more refined and complex molecular genetic techniques. In this chapter, we first discuss some of the conceptual and technical aspects of the gene transfer to the nervous system (NS), in order to provide a framework for the discussion of alternative approaches to gene therapy for neurological diseases. Although we take a broad approach, it is not our purpose to review extensively all aspects of gene therapy in the NS, but rather to select a few examples that can serve to illustrate the potential, the problems, and some of the possible strategies that may bring us closer to the goal of effective and safe gene therapy for NS disorders. As a complement, the reader is referred to several recent reviews (and the work cited therein) that have already dealt with the details of some of the important breakthroughs in this field during recent years (Fisher and Ray 1994; Crystal 1995; Karpati et al. 1996; Snyder and Fisher 1996; Doering 1997; Freese 1997; Friedmann 1997; Lanza et al. 1996; Vile 1997; Ho and Sapolski 1997; Kay et al. 1997; Miller and Whelan 1997; Raymon et al. 1997; Verma and Somia 1997).

In this brief introduction, we would also like to make some comments...