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Regulated Gene Therapy.

Breger, Ludivine LU ; Elgstrand, Erika LU ; Quintino, Luis LU and Lundberg, Cecilia LU (2016) In Methods in Molecular Biology 1382. p.57-66
Abstract
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... (More)
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. (Less)
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author
organization
publishing date
type
Contribution to journal
publication status
published
subject
in
Methods in Molecular Biology
volume
1382
pages
57 - 66
publisher
Springer
external identifiers
  • pmid:26611578
  • scopus:84948808180
ISSN
1940-6029
DOI
10.1007/978-1-4939-3271-9_4
language
English
LU publication?
yes
id
c25daa22-f956-4bf0-9b2b-5525c22da489 (old id 8234430)
alternative location
http://www.ncbi.nlm.nih.gov/pubmed/26611578?dopt=Abstract
date added to LUP
2015-12-01 16:21:09
date last changed
2017-05-07 04:25:11
@article{c25daa22-f956-4bf0-9b2b-5525c22da489,
  abstract     = {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.},
  author       = {Breger, Ludivine and Elgstrand, Erika and Quintino, Luis and Lundberg, Cecilia},
  issn         = {1940-6029},
  language     = {eng},
  pages        = {57--66},
  publisher    = {Springer},
  series       = {Methods in Molecular Biology},
  title        = {Regulated Gene Therapy.},
  url          = {http://dx.doi.org/10.1007/978-1-4939-3271-9_4},
  volume       = {1382},
  year         = {2016},
}