Microglial replacement strategy to treat microgliopathy
Colony-stimulating factor 1 receptor (CSF1R) gene mutation (I794T) is linked to primary microgliopathy manifesting as leukoencephalopathy.
The researchers define the clinical features of patients carrying the CSF1R p.I794T variant and establish a corresponding knockin mouse model.
The authors demonstrate that knockin mice exhibited hallmark features of CSF1R-related disorder (CSF1R-RD).
They show that Csf1rI792T/+ microglia adopt a disease associated state and that a microglial replacement strategy termed “duplicate-cyclic microglial depletion for transplantation” (DCMDT), mitigates cognitive and neuropathological deficits in CSF1R-RD.
https://www.cell.com/cell-reports-medicine/fulltext/S2666-3791(26)00058-3
