Clinical applications involving CNS gene transfer

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

Research output: Contribution to journalArticle

28 Citations (Scopus)

Abstract

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
Volume87
DOIs
StatePublished - Jan 1 2014

Fingerprint

Central Nervous System Diseases
Genetic Therapy
Central Nervous System
Dependovirus
Viral Genes
Drug Delivery Systems
Blood-Brain Barrier
Nervous System
Genes
Proteins
Clinical Trials
Pharmacology
Therapeutics

Keywords

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

ASJC Scopus subject areas

  • Genetics

Cite this

Clinical applications involving CNS gene transfer. / Kantor, Boris; McCown, Thomas; Leone, Paola; Gray, Steven J.

In: Advances in Genetics, Vol. 87, 01.01.2014, p. 71-124.

Research output: Contribution to journalArticle

Kantor, Boris ; McCown, Thomas ; Leone, Paola ; Gray, Steven J. / Clinical applications involving CNS gene transfer. In: Advances in Genetics. 2014 ; Vol. 87. pp. 71-124.
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