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Novel disease-specific promoters for use in gene therapy for Parkinson's disease.

Elgstrand, Erika LU ; Gussing, Fredrik LU ; Quintino, Luis LU and Lundberg, Cecilia LU (2012) In Neuroscience Letters 530(1). p.29-34
Abstract
Gene therapy is a promising therapeutic tool for Parkinson's disease (PD), but there is a lack of evaluated cell specific promoters that are relevant for the disease. We have chosen PD relevant promoter candidates for gene therapy vectors based on either previous studies; Drd1a, Drd2 and pDyn, or from a microarray study on parkinsonian patients; ACE, DNAJC3, GALNS, MAP1a and RNF25. These candidates have been evaluated in rat striatum to determine their suitability for use in cell specific vectors. The promoters had a neuronal specificity of 91-100%. The efficiency of the promoters was variable, but RNF25, DNAJC3 and MAP1a were comparable to widely used ubiquitous promoters. MAP1a was also affected by dopamine depletion.
Please use this url to cite or link to this publication:
author
organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
Promoter, Parkinson's disease, Gene therapy, Neuron-specific
in
Neuroscience Letters
volume
530
issue
1
pages
29 - 34
publisher
Elsevier
external identifiers
  • wos:000311664000006
  • pmid:23063686
  • scopus:84868301718
ISSN
0304-3940
DOI
10.1016/j.neulet.2012.09.059
language
English
LU publication?
yes
id
39771ee8-f471-417a-9f7a-905468f9ee0f (old id 3160760)
alternative location
http://www.ncbi.nlm.nih.gov/pubmed/23063686?dopt=Abstract
date added to LUP
2012-11-01 16:29:53
date last changed
2017-06-04 03:05:07
@article{39771ee8-f471-417a-9f7a-905468f9ee0f,
  abstract     = {Gene therapy is a promising therapeutic tool for Parkinson's disease (PD), but there is a lack of evaluated cell specific promoters that are relevant for the disease. We have chosen PD relevant promoter candidates for gene therapy vectors based on either previous studies; Drd1a, Drd2 and pDyn, or from a microarray study on parkinsonian patients; ACE, DNAJC3, GALNS, MAP1a and RNF25. These candidates have been evaluated in rat striatum to determine their suitability for use in cell specific vectors. The promoters had a neuronal specificity of 91-100%. The efficiency of the promoters was variable, but RNF25, DNAJC3 and MAP1a were comparable to widely used ubiquitous promoters. MAP1a was also affected by dopamine depletion.},
  author       = {Elgstrand, Erika and Gussing, Fredrik and Quintino, Luis and Lundberg, Cecilia},
  issn         = {0304-3940},
  keyword      = {Promoter,Parkinson's disease,Gene therapy,Neuron-specific},
  language     = {eng},
  number       = {1},
  pages        = {29--34},
  publisher    = {Elsevier},
  series       = {Neuroscience Letters},
  title        = {Novel disease-specific promoters for use in gene therapy for Parkinson's disease.},
  url          = {http://dx.doi.org/10.1016/j.neulet.2012.09.059},
  volume       = {530},
  year         = {2012},
}