Gene therapy approaches in central nervous system regenerative medicine

This chapter introduces the use of gene therapy strategies as therapeutic approaches for regenerative medicine of diseases affecting the central nervous system (CNS). It starts by defining gene therapy and its potential, followed by a general description of the most used vectors in clinical assays as well as their relative advantages and disadvantages. Then, there is a section describing the main vectors used in preclinical and clinical assays in CNS, their tropisms and the recommended administration routes to achieve restricted or widespread vector distribution, or to target specific cell types. Finally, there is a summary of neurodegenerative diseases currently treated by gene therapy strategies, the success and pitfalls reported at present.