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Neurosci Lett. 2012 Nov 14;530(1):29-34. doi: 10.1016/j.neulet.2012.09.059. Epub 2012 Oct 12.

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

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CNS Gene Therapy Unit, BMC A11, Dept. of Experimental Medical Science, Lund University, S-221 84 Lund, Sweden.


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.

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