Engineered stem cell-derived microglia as therapeutic vehicle for experimental autoimmune encephalomyelitis
17.01.2013
Beutner C, Lepperhof V, Dann A, Linnartz-Gerlach B, Litwak S, Napoli I, Prinz M, Neumann H.
?Gene Ther. 2013;20(8):797-806
Inflammation can be prevented in most inflammatory brain diseases, while tissue repair of the lesioned central nervous system (CNS) is still a major challenge. The CNS is difficult to access for protein therapeutics due to the blood-brain barrier. Here, we show that genetically engineered embryonic stem cell-derived microglia (ESdM) are a suitable therapeutic vehicle for neurotrophin-3 (NT3) in experimental autoimmune encephalomyelitis (EAE). The intravenously transplanted ESdM migrated into the inflammatory CNS lesions and engrafted there as microglial cells. EAE afflicted mice treated with ESdM that were genetically modified to express NT3 showed stable recovery from disease symptoms.
