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

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 t...

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Detalles Bibliográficos
Autores principales: Naidoo, Jerusha, Young, Deborah
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Hindawi Publishing Corporation 2012
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3261487/
https://www.ncbi.nlm.nih.gov/pubmed/22272373
http://dx.doi.org/10.1155/2012/595410
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author Naidoo, Jerusha
Young, Deborah
author_facet Naidoo, Jerusha
Young, Deborah
author_sort Naidoo, Jerusha
collection PubMed
description 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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spelling pubmed-32614872012-01-23 Gene Regulation Systems for Gene Therapy Applications in the Central Nervous System Naidoo, Jerusha Young, Deborah Neurol Res Int Review Article 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. Hindawi Publishing Corporation 2012 2012-01-05 /pmc/articles/PMC3261487/ /pubmed/22272373 http://dx.doi.org/10.1155/2012/595410 Text en Copyright © 2012 J. Naidoo and D. Young. https://creativecommons.org/licenses/by/3.0/ This is an open access article distributed under the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
spellingShingle Review Article
Naidoo, Jerusha
Young, Deborah
Gene Regulation Systems for Gene Therapy Applications in the Central Nervous System
title Gene Regulation Systems for Gene Therapy Applications in the Central Nervous System
title_full Gene Regulation Systems for Gene Therapy Applications in the Central Nervous System
title_fullStr Gene Regulation Systems for Gene Therapy Applications in the Central Nervous System
title_full_unstemmed Gene Regulation Systems for Gene Therapy Applications in the Central Nervous System
title_short Gene Regulation Systems for Gene Therapy Applications in the Central Nervous System
title_sort gene regulation systems for gene therapy applications in the central nervous system
topic Review Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3261487/
https://www.ncbi.nlm.nih.gov/pubmed/22272373
http://dx.doi.org/10.1155/2012/595410
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