Adeno-associated virus and lentivirus vectors: a refined toolkit for the central nervous system

•AAV and LV vectors for gene delivery in the central nervous system.•Gene therapy as a major driver for viral vector development.•Treatment of neurodegenerative diseases with gene therapies.•Cell-type specific transgene expression.

The last two decades have witnessed the increasing instrumentalization of viruses, which have progressively evolved into highly potent gene transfer vehicles for a wide spectrum of applications. In the context of the central nervous system (CNS), their unique gene delivery features and targeting specificities have been exploited not only to improve our understanding of basic neurobiology, but also to investigate diseases or deliver therapeutic candidates. As a result, we have started moving away from the opportunistic use of recombinant vectors that are derived from naturally existing viruses toward the rational engineering of tailored lentivirus (LV) and adeno-associated virus (AAV) vectors for specific use in the CNS.