Sex- and region-biased depletion of microglia/macrophages attenuates CLN1 disease in mice.

ABSTRACT:

Background

The neuronal ceroid lipofuscinoses (CLN diseases) are fatal lysosomal storage diseases causing neurodegeneration in the CNS. We have previously shown that neuroinflammation comprising innate and adaptive immune reactions drives axonal damage and neuron loss in the CNS of palmitoyl protein thioesterase 1-deficient (Ppt1^-/-) mice, a model of the infantile form of the diseases (CLN1). Therefore, we here explore whether pharmacological targeting of innate immune cells modifies disease outcome in CLN1 mice.

Methods

We applied treatment with PLX3397 (150 ppm in the chow), a potent inhibitor of the colony stimulating factor-1 receptor (CSF-1R) to target innate immune cells in CLN1 mice. Experimental long-term treatment was non-invasively monitored by longitudi

SUBMITTER: Berve K 

PROVIDER: S-EPMC7594417 | biostudies-literature | 2020 Oct

REPOSITORIES: biostudies-literature