Science Spotlight: Microglial transplantation for ALSP, AlaMab’s osteosarcoma mAb and more

A pair of companion Neuron papers presented a new mouse model of adult-onset leukoencephalopathy with axonal spheroids and pigmented glia that recapitulates human disease pathologies better than prior models, and used it to show the therapeutic potential of microglial transplantation. 

A University of California Irvine team developed the mouse model, which lacks the fms-intronic regulatory element (FIRE) enhancer that regulates the expression of CSF1R, mutations in which cause ALSP. In four-month-old mice, transplantation of patient induced pluripotent stem cell (iPSC)-derived microglia with CRISPR-corrected ALSP reversed pre-existing spheroids, astrogliosis and calcification...