Wolfe Laboratory

The Wolfe Laboratory works on transferring disease-correcting genes into the central nervous system (CNS) in animal models of human genetic diseases. In these diseases, the CNS is often not rescued by the therapies that help the rest of the body. The lab studies both ex vivo gene transfer into neural stem cells that are then transplanted and in vivo transfer using vectors injected directly into the brain.

Wolfe Laboratory studies involve comparisons of both the vectors used to introduce the genes into cells and the properties of the genes themselves. Additionally, we examine the ability of different cell types and subregions of the brain to be corrected. We also pursue new methods to follow the corrected cells and the expression of the correcting gene in the live animal using MRI and PET techniques. For a complete understanding of the therapy, we also are working on achieving a better understanding of the mechanism of these diseases in the brain.

Wolfe Laboratory projects involve:

• The molecular design and engineering of vectors
• The understanding of the fate of vector-transferred genes in the brain
• The regulation of gene expression from vectors
• The biology of neural stem cells
• The study of induced pluripotent stem cells (iPS)
• The use of imaging studies in genetic disease and gene therapy, and the proteomic and genomics analysis of the neurodegenerative brain.

For students, projects can be tailored to interests, learning goals, and experience.