Abstract
Gene therapy has been in the medical spotlight for over a decade now and its enormous potential offers hope to many sufferers of diseases involving a genetic component. Although progress has been made, clinical gene therapy is still quite a remote possibility especially in the relatively new field of targeting the central nervous system (CNS). The treatment of CNS disorders by gene therapy offers unique therapeutic challenges due to the fragility and complexity of the brain; delivery through the protective envelope of the brain is associated with many risks, and gene transfer into post-mitotic neurons is restrictive. Nevertheless, we believe that neurological gene therapy still has good future prospects. This short review will attempt to describe current gene therapeutic strategies, delivery methods and the most promising vectors for use in neurodegenerative disorders. We also provide a short description of our own system, currently under development.
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