Clinical applications involving CNS gene transfer

Boris Kantor, Thomas McCown, Paola Leone, Steven J. Gray

Research output: Contribution to journalArticlepeer-review

44 Scopus citations


Diseases of the central nervous system (CNS) have traditionally been the most difficult to treat by traditional pharmacological methods, due mostly to the blood-brain barrier and the difficulties associated with repeated drug administration targeting the CNS. Viral vector gene transfer represents a way to permanently provide a therapeutic protein within the nervous system after a single administration, whether this be a gene replacement strategy for an inherited disorder or a disease-modifying protein for a disease such as Parkinson's. Gene therapy approaches for CNS disorders has evolved considerably over the last two decades. Although a breakthrough treatment has remained elusive, current strategies are now considerably safer and potentially much more effective. This chapter will explore the past, current, and future status of CNS gene therapy, focusing on clinical trials utilizing adeno-associated virus and lentiviral vectors.

Original languageEnglish (US)
Pages (from-to)71-124
Number of pages54
JournalAdvances in Genetics
StatePublished - Jan 1 2014


  • AAV
  • Canavan disease
  • Clinical trial
  • CNS
  • Gene therapy
  • Lentivirus
  • Parkinson's disease
  • Retrovirus
  • Vector

ASJC Scopus subject areas

  • Genetics


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