title: Methods for gene transfer to the central nervous system.
abstract: Gene transfer is an increasingly utilized approach for research and clinical applications involving the central nervous system (CNS). Vectors for gene transfer can be as simple as an unmodified plasmid, but more commonly involve complex modifications to viruses to make them suitable gene delivery vehicles. This chapter will explain how tools for CNS gene transfer have been derived from naturally occurring viruses. The current capabilities of plasmid, retroviral, adeno-associated virus, adenovirus, and herpes simplex virus vectors for CNS gene delivery will be described. These include both focal and global CNS gene transfer strategies, with short- or long-term gene expression. As is described in this chapter, an important aspect of any vector is the cis-acting regulatory elements incorporated into the vector genome that control when, where, and how the transgene is expressed.
authors.affiliation: Department of Pharmacology, Physiology, and Neuroscience, University of South Carolina, Columbia, SC, USA.
authors.forename: Boris
authors.initials: B
authors.lastname: Kantor
authors.affiliation: Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
authors.forename: Rachel M
authors.initials: RM
authors.lastname: Bailey
authors.affiliation: Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
authors.forename: Keon
authors.initials: K
authors.lastname: Wimberly
authors.affiliation: Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
authors.forename: Sahana N
authors.initials: SN
authors.lastname: Kalburgi
authors.affiliation: 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.
authors.forename: Steven J
authors.initials: SJ
authors.lastname: Gray
mesh_terms: D000256:Adenoviridae / Q000235:genetics; D002490:Central Nervous System / Q000821:virology; D015870:Gene Expression / Q000235:genetics; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods; D005822:Genetic Vectors / Q000627:therapeutic use*; D006801:Humans; D016086:Lentivirus / Q000235:genetics; D018139:Simplexvirus / Q000235:genetics
keywords: AAV; Adenovirus; CNS; Gene therapy; Lentivirus; Plasmid; Promoter; Vector