University of Helsinki
Using novel 3UTR targeting based approaches to study gene function and treat neurodegenerative disease
Academy of Finland
Brain function depends on neuronal networks built and maintained in part via accurate spatiotemporal expression of neurotrophic factors (NTFs). In neurodegenerative diseases such as Parkinsons disease, contacts between specific neurons are lost. In theory, elevation of NTFs in the correct target neurons would enable restoration of the original neuronal network connections. However, currently no means to specifically elevate the levels of NTFs limited to the correct expression site are available. By identifying molecules responsible for regulation of endogenous NTF levels and developing new genetics tools we aim to show that restoration of neural networks is possible. The new tools already generated in my laboratory or under construction provide a new way to study gene function, have a potential to reveal novel drug targets and provide insight into how NTFs regulate specific neuronal network function.