Lentiviral Vectors for Gene Transfer to the Central Nervous System: Applications in Lysosomal Storage Disease Animal Models

Recombinant viral vectors have been used to study a variety of fundamental issues in developmental neurobiology, as well as pathogenesis and treatments for various neurodegenerative diseases. Lentiviral vectors are valuable tools for neurobiology research, because of their ability to transduce nondividing cells such as neurons, to introduce new genes into central nervous system (CNS) cells in vivo and in vitro. In this chapter, we provide a variety of detailed methods for evaluating lentiviral transduction of the CNS in the mucopolysaccharidosis type VII (MPS VII) mouse model.