Manipulation of Gene Expression in the Central Nervous System with Lentiviral Vectors

Viral vector-mediated gene transfer is widely used to manipulate gene expression (overexpression or knock down) in cultures and in different tissues of animals. Vectors based on lentiviruses have particularly useful features. Lentiviral vectors mediate gene transfer into any neuronal cell types and induce sustained expression without significant immune responses after delivery into the nervous system. Lentivirus-mediated expression of therapeutic genes has led to long-term treatment of animal models of neurological disorders, such as spinal injury, Parkinson’s disease, Huntington’s disease, and Alzheimer’s disease. Here, we describe the preparation and purification of lentiviral vectors and methods of lentiviral infection in primary neural cultures and in brain regions of interest by stereotaxic injection.