CNS Gene Therapy

Organisational unit: Research group

Research areas and keywords

UKÄ subject classification

  • Neurosciences
  • Medical Biotechnology


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

Elsa Westerlund, Valfridsson, C., Yi, D. X. & Jenny J. Persson, 2019 Jun 18, In : Frontiers in Immunology. 10, JUN, 1385.

Research output: Contribution to journalArticle

De Luca, M., Aiuti, A., Cossu, G., Malin Parmar, Pellegrini, G. & Robey, P. G., 2019, In : Nature Cell Biology. 21, 7, p. 801-811 11 p.

Research output: Contribution to journalReview article

Luis Quintino, Martino Avallone, Brännstrom, E., Kavanagh, P., Marcus Lockowandt, Garcia Jareño, P., Breger, L. S. & Cecilia Lundberg, 2019, In : Gene Therapy. 26, 1-2, p. 57-64

Research output: Contribution to journalArticle

View All (66)