CNS Gene Therapy

Organisational unit: Research group

Research

In the gene therapy group our main interest is to develop systems for sufficient and sustained expression of transgenes in the CNS. We are using gene transfer to express trophic factors or neurotransmitter-producing enzymes in animal models of neurodegenerative disease. We are currently working with three different vector systems: adeno-associated viral, retroviral and lentiviral. These vectors can be used both for direct injection (in vivo gene transfer) or transduction of cells in culture for subsequent transplantation (ex vivo gene transfer). To address the problem of sustained expression in vivo (a major limitation of many gene transfer methods as of now) we are investigating the use of different promoters, post-transcriptional regulating elements and vectors.

Recent research outputs

Chtarto, A., Humbert-Claude, M., Bockstael, O., Das, A. T., Boutry, S., Ludivine S Breger, Klaver, B., Melas, C., Barroso-Chinea, P., Gonzalez-Hernandez, T., Muller, R. N., DeWitte, O., Levivier, M., Cecilia Lundberg, Berkhout, B. & Tenenbaum, L. 2016 In : Molecular therapy. Methods & clinical development. 5, 16027

Research output: Contribution to journalArticle

Ludivine Breger, Elgstrand, E., Luis Quintino & Cecilia Lundberg 2016 In : Methods in Molecular Biology. 1382, p. 57-66

Research output: Contribution to journalArticle

Elgstrand, E., Luis Quintino, Manfre, G. & Cecilia Lundberg 2014 Viral Vector Approaches in Neurobiology and Brain Diseases . Brambilla, R. (ed.). Springer, Vol. 82, p. 181-191 11 p. 10

Research output: Chapter in Book/Report/Conference proceedingBook chapter

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