Vector-mediated gene regulation in midbrain dopamine neurons

摘要

The present invention provides compositions and methods for vector mediated gene regulation in neurons. Specifically, the present invention provides therapeutic compositions comprising viral vectors that allow for the over-expression and RNAi mediated knockdown of genes in vivo. The present invention further provides methods for treating or preventing neurodegeneration in a subject, and for protecting neurons from damage in the context of neurodegenerative disorders. Additionally, the present invention provides a composition, and use of the composition in improving animal models of neurodegeneration.