Intraventricular transplantation of oligodendrocyte progenitors into a fetal myelin mutant results in widespread formation of myelin

Transplantation of myelin-forming cells is a promising strategy far the tre atment of myelin disorders. In this study, transplantation of glial cell pr ogenitors into the cerebral ventricles of the embryonic myelin-deficient ra t, a model of Pelizaeus-Merzbacher disease, was performed to assess the abi lity of these cells to incorporate into the developing brain and produce my elin. The donor cells migrated into the white and gray matter and produced myelin at widespread sites ranging from the corpus callosum and optic nerve to the cerebellum. These data suggest that myelin repair might be achieved by intraventricular delivery and transependymal incorporation of myelin-pr oducing cells. Because these cells were genetically transduced to express a reporter gene, similar ex vivo manipulation with genes known to promote su rvival, migration, or proliferation of the transplanted cells could be used to enhance repair. Such a therapeutic strategy may be feasible in patients with inherited myelin disorders or in multiple sclerosis, particularly whe re the lesions are periventricular.