title: Gene regulation systems for gene therapy applications in the central nervous system.
abstract: Substantial progress has been made in the development of novel gene therapy strategies for central nervous system (CNS) disorders in recent years. However, unregulated transgene expression is a significant issue limiting human applications due to the potential side effects from excessive levels of transgenic protein that indiscriminately affect both diseased and nondiseased cells. Gene regulation systems are a tool by which tight tissue-specific and temporal regulation of transgene expression may be achieved. This review covers the features of ideal regulatory systems and summarises the mechanics of current exogenous and endogenous gene regulation systems and their utility in the CNS.
authors.affiliation: Department of Pharmacology and Clinical Pharmacology and Centre for Brain Research, University of Auckland, Auckland 1023, New Zealand.
authors.forename: Jerusha
authors.initials: J
authors.lastname: Naidoo
authors.forename: Deborah
authors.initials: D
authors.lastname: Young