Clinical applications involving CNS gene transfer.

[canavan disease]

Diseases of the central nervous system (CNS ) have traditionally been the most difficult to treat by traditional pharmacological methods , due mostly to the blood -brain barrier and the difficulties associated with repeated drug administration targeting the CNS . Viral vector gene transfer represents a way to permanently provide a therapeutic protein within the nervous system after a single administration , whether this be a gene replacement strategy for an inherited disorder or a disease-modifying protein for a disease such as Parkinson 's . Gene therapy approaches for CNS disorders has evolved considerably over the last two decades . Although a breakthrough treatment has remained elusive , current strategies are now considerably safer and potentially much more effective . This chapter will explore the past , current , and future status of CNS gene therapy , focusing on clinical trials utilizing adeno-associated virus and lentiviral vectors .