Regulated Gene Therapy.

Ludivine Breger, Erika Elgstrand Wettergren, Luis Quintino, Cecilia Lundberg
Gene Therapy for Neurological Disorders. 2016-01-01; : 57-66
DOI: 10.1007/978-1-4939-3271-9_4

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1. Methods Mol Biol. 2016;1382:57-66. doi: 10.1007/978-1-4939-3271-9_4.

Regulated Gene Therapy.

Breger L(1), Wettergren EE(1), Quintino L(1), Lundberg C(2).

Author information:
(1)Department of Experimental Medical Sciences, CNS Gene Therapy Unit, Wallenberg
Neuroscience Center, Lund University, BMC A11, 221 84, Lund, Sweden.
(2)Department of Experimental Medical Sciences, CNS Gene Therapy Unit, Wallenberg
Neuroscience Center, Lund University, BMC A11, 221 84, Lund, Sweden.
.

Gene therapy represents a promising approach for the treatment of monogenic and
multifactorial neurological disorders. It can be used to replace a missing gene
and mutated gene or downregulate a causal gene. Despite the versatility of gene
therapy, one of the main limitations lies in the irreversibility of the process:
once delivered to target cells, the gene of interest is constitutively expressed
and cannot be removed. Therefore, efficient, safe and long-term gene modification
requires a system allowing fine control of transgene expression.Different systems
have been developed over the past decades to regulate transgene expression after
in vivo delivery, either at transcriptional or post-translational levels. The
purpose of this chapter is to give an overview on current regulatory system used
in the context of gene therapy for neurological disorders. Systems using external
regulation of transgenes using antibiotics are commonly used to control either
gene expression using tetracycline-controlled transcription or protein levels
using destabilizing domain technology. Alternatively, specific promoters of genes
that are regulated by disease mechanisms, increasing expression as the disease
progresses or decreasing expression as disease regresses, are also examined.
Overall, this chapter discusses advantages and drawbacks of current molecular
methods for regulated gene therapy in the central nervous system.

DOI: 10.1007/978-1-4939-3271-9_4
PMID: 26611578 [Indexed for MEDLINE]

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