Viral vector gene delivery to the brain for treating neurogenetic diseases

Abstract

Treatment of neurogenetic diseases is often difficult, as potential treatment must correct pathology that is globally present. A promising approach to treatment of these types of diseases is gene therapy, which employs the use of viral vectors to deliver a therapeutic gene. The three most commonly used viral vectors, herpes simplex viruses (HSVs), lentiviruses (LVs) and adeno-associated viruses (AAVs) are described, as well as the potential routes of administration for delivery of viral vectors in humans. This chapter highlights the main neurogenetic diseases for which viral vector-based therapies are being developed.