Demonstrates neuroprotective benefits of depleting retinal mononuclear phagocytes in mice with mitochondrial optic neuropathy, suggesting potential adjunct therapies.
Pexidartinib effectively depletes retinal MNPs and is neuroprotective in the setting of severe RGC mitochondrial dysfunction. This therapeutic effect is additive to that of hypoxia. Combating retinal neuro-inflammation may therefore be a useful adjunct therapy in mitochondrial optic neuropathies like Leber hereditary optic neuropathy (LHON).
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Smolar et al. (2026) studied this question.
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