Leukodystrophies represent a wide variety of hereditary disorders of the white matter in the central nervous system, where the patients, mostly in infancy or childhood, suffer from progressive and often fatal neurological symptoms due to either a delay or lack of myelin development or loss of myelin. As only supportive therapies are available for the majority of the leukodystrophies, replacing genetically defective oligodendrocytes with intact oligodendrocytes by transplantation has a potential as a curative therapy. Animal models of leukodystrophies have been valuable in developing effective strategies of myelin repair in human diseases. This chapter discusses the animal models of leukodystrophies and describes methods for (a) derivation of mouse oligodendrocyte progenitor cells (OPCs) in vitro as a source of donor myelin-forming cells and (b) transplantation of OPCs into the brain and spinal cord of mouse models of leukodystrophies.
