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.

Original languageEnglish
Title of host publicationHandbook of Innovations in Central Nervous System Regenerative Medicine
Number of pages24
ISBN (Electronic)9780128180846
Publication statusPublished - 1 Jan 2020


  • Adenoassociated vectors
  • Central nervous system
  • Clinical assays
  • Gene therapy
  • Lentivirus
  • Regenerative medicine


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