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.
Copyright © 2012 Elsevier Ireland Ltd. All rights reserved.
Publication types
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Research Support, Non-U.S. Gov't
MeSH terms
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Animals
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Chondroitinsulfatases / genetics
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Corpus Striatum / physiology
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Dopamine / deficiency
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Dopaminergic Neurons / physiology
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Gene Transfer Techniques*
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Genetic Therapy / methods*
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HSP40 Heat-Shock Proteins / genetics
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Humans
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Lentivirus / genetics*
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Microtubule-Associated Proteins / genetics
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Oligonucleotide Array Sequence Analysis
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Parkinson Disease / genetics*
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Parkinson Disease / therapy*
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Peptidyl-Dipeptidase A / genetics
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Promoter Regions, Genetic / genetics*
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Rats
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Rats, Sprague-Dawley
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Ubiquitin-Protein Ligases / genetics
Substances
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DNAJC3 protein, human
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HSP40 Heat-Shock Proteins
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MAP1A protein, human
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Microtubule-Associated Proteins
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RNF25 protein, human
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Ubiquitin-Protein Ligases
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Chondroitinsulfatases
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GALNS protein, human
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ACE protein, human
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Peptidyl-Dipeptidase A
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Dopamine