CNS Gene Therapy

Our 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 regulated and cell-specific expression in vivo (a major challenge of many gene transfer methods as of now) we are investigating the use of different promoters, post-transcriptional regulating elements, and vectors.

Aims

To develop cell-specific lentiviral vectors that can be regulated and used to treat the hallmarks features of Parkinson’s disease.
Investigate the differential expression of miRNAs in subpopulations of neurons in the striatum in different states of dopamine signaling status.
Using transgene regulation we attempt to reinstate the disease-modifying GDNF response in nigral neurons in models of Parkinson’s disease.
Use CRISPR/Cas 9 systems to regulate the expression of key factors that have been suggested to be important in the disease process in Parkinson’s disease.

Impact

A major challenge for molecular medicine is to be innovative, truly superior to and more precise than traditional paradigms. We aim to achieve these objectives using a model of Parkinson’s disease.There is currently no cure for Parkinson’s disease. If our research is successful, we will get one step closer to a cure and will also have gained new knowledge about neurodegeneration and how to counter-act it.