Gene Regulation Systems for Gene Therapy Applications in the Central Nervous System | Zendy

Jerusha Naidoo | Zendy; Deborah Young | Zendy

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Gene Regulation Systems for Gene Therapy Applications in the Central Nervous System

Author(s) -

Jerusha Naidoo,

Deborah Young

Publication year - 2012

Publication title -

neurology research international

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 0.365

H-Index - 31

eISSN - 2090-1852

pISSN - 2090-1860

DOI - 10.1155/2012/595410

Subject(s) - transgene , genetic enhancement , gene , limiting , gene expression , central nervous system , medicine , endogeny , neuroscience , regulation of gene expression , genetically modified mouse , computational biology , bioinformatics , biology , genetics , mechanical engineering , engineering

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.

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