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Adv Genet. 2014;87:71-124. doi: 10.1016/B978-0-12-800149-3.00002-0.

Clinical applications involving CNS gene transfer.

Author information

1
Department of Pharmacology, Physiology, and Neuroscience, University of South Carolina, Columbia, SC, USA.
2
Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA; Department of Psychiatry, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
3
Department of Cell Biology, Rowan University, Camden, NJ, USA.
4
Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA; Department of Ophthalmology, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.

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.

KEYWORDS:

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

[Indexed for MEDLINE]
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