Neurodegenerative diseases (NDs) have a profound impact on human health worldwide\nand their incidence is predicted to increase as the population ages. ND severely limits the quality\nof life and leads to early death. Aside from treatments that may reduce symptoms, NDs are almost\ncompletely without means of therapeutic intervention. The genetic and biochemical basis of many\nNDs is beginning to emerge although most have complex etiologies for which common themes remain\npoorly resolved. Largely relying on progress in vector design, gene therapy is gaining increasing\nsupport as a strategy for genetic treatment of diseases. Here we describe recent developments in the\nengineering of highly defective herpes simplex virus (HSV) vectors suitable for transfer and long-term\nexpression of large and/or multiple therapeutic genes in brain neurons in the complete absence of\nviral gene expression. These advanced vector platforms are safe, non-inflammatory, and persist in\nthe nerve cell nucleus for life. In the near term, it is likely that HSV can be used to treat certain NDs\nthat have a well-defined genetic cause. As further information on disease etiology becomes available,\nthese vectors may take on an expanded role in ND therapies, including gene editing and repair.
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