Curative therapies still do not exist for most CNS diseases but gene therapy is a promising new approach. We propose that it will be possible to modify brain function and pathophysiology by targeted delivery of specific curative factors to selected populations of brain cells that are affected by disease. This opens the door for effective treatment regimes, which can be tailored to individual patients needs. However, currently available gene transfer vectors have limitations regarding safety and efficacy, as they do not allow for targeting of specific populations of neurons or glia or regulation of transgene expression.

The NEUGENE consortium has been founded by leading European scientists from academia and industry to overcome these limitations. The consortium will develop Adeno-associated virus (AAV) and Lentivirus (LV) - based tools for targeted and regulated gene transfer into different populations of CNS cells. The consortium will provide a selection of vectors that are optimized for different therapeutic approaches, e.g. regulated expression of neurotrophic factors or manipulation of neurotransmitter synthesis in specific neurons.

NEUGENE has three major goals: 1) targeting gene transfer vectors to specific populations of neurons and glia, by transcriptional regulation and miRNA-mediated de-targeting and by exploiting the cell-specific tropism of novel types of viral vectors, 2) tight control over expression levels of therapeutic genes by using regulated systems based on different principles, and 3) establishing the safety of the novel vector tools.

NEUGENE will verify the functional efficacy of the novel CNS gene transfer tools in a well-established animal model of Parkinson's Disease (PD). This disorder affects over 1.000.000 Europeans and is increasing in prevalence with the aging population. Importantly,  principles and mechanisms developed and evaluated within the consortium will also be of direct relevance for gene therapy of many other brain disorders.