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

Summary, in English

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.

• Författare: Erika Elgstrand; Fredrik Gussing; Luis Quintino; Cecilia Lundberg
• Avdelning/ar: CNS Genterapi; MultiPark: Multidisciplinary research focused on Parkinson´s disease
• Publiceringsår: 2012
• Språk: Engelska
• Sidor: 29-34
• Publikation/Tidskrift/Serie: Neuroscience Letters
• Volym: 530
• Issue: 1
• Länkar: http://www.ncbi.nlm.nih.gov/pubmed/23063686?dopt=Abstract; http://dx.doi.org/10.1016/j.neulet.2012.09.059
• Dokumenttyp: Artikel i tidskrift
• Förlag: Elsevier
• Ämne: Neurosciences
• Nyckelord: Promoter; Parkinson's disease; Gene therapy; Neuron-specific
• Status: Published
• Forskningsgrupp: CNS Gene Therapy
• ISBN/ISSN/Övrigt: ISSN: 0304-3940