Pluripotent stem cell-derived neurons for transplantation in Huntington's disease.

Pluripotent stem cells present a potentially unlimited source of cells for regenerative medicine, providing that they can be efficiently and accurately differentiated to the target cell type. The principle target cell for Huntington's disease is the striatal medium spiny neuron. In this chapter, we review strategies for directing medium spiny neuron differentiation, based on known developmental principles, and we discuss the remaining hurdles on the road to engineering such cells for therapeutic application in Huntington's disease.