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Curcumin therapy in a Plp1 transgenic mouse model of Pelizaeus-Merzbacher disease
Dirk B Epplen1, Thomas Prukop2, Tobias Nientiedt1
1Department of Neurogenetics, Max-Planck-Institute of Experimental Medicine Göttingen, Germany.
Objective:
Pelizaeus-Merzbacher disease (PMD) is a progressive and lethal leukodystrophy caused by mutations affecting the proteolipid protein (PLP1) gene. The most common cause of PMD is a duplication of PLP1 and at present there is no curative therapy available.
Methods:
By using transgenic mice carrying additional copies of Plp1, we investigated whether curcumin diet ameliorates PMD symptoms. The diet of Plp1 transgenic mice was supplemented with curcumin for 10 consecutive weeks followed by phenotypical, histological and immunohistochemical analyses of the central nervous system. Plp1 transgenic and wild-type mice fed with normal chow served as controls.
Results:
Curcumin improved the motor phenotype performance of Plp1 transgenic mice by 50% toward wild-type level and preserved myelinated axons by 35% when compared to Plp1 transgenic controls. Furthermore, curcumin reduced astrocytosis, microgliosis and lymphocyte infiltration in Plp1 transgenic mice. Curcumin diet did not affect the pathologically increased Plp1 mRNA abundance. However, high glutathione levels indicating an oxidative misbalance in the white matter of Plp1 transgenic mice were restored by curcumin treatment.
Interpretation:
Curcumin may potentially serve as an antioxidant therapy of PMD caused by PLP1 gene duplication.
Insights
Curcumin supplementation improved motor function and preserved myelinated axons in Pelizaeus-Merzbacher disease (PMD) mouse models. This antioxidant therapy shows promise for treating PMD caused by PLP1 gene duplication.
Area of Science:
- Neuroscience
- Genetics
- Pharmacology
Background:
- Pelizaeus-Merzbacher disease (PMD) is a fatal leukodystrophy.
- Mutations in the proteolipid protein 1 (PLP1) gene, often duplications, cause PMD.
- Currently, no curative treatments exist for PMD.
Purpose of the Study:
- To investigate the therapeutic potential of curcumin in a mouse model of PMD.
- To determine if curcumin diet ameliorates PMD symptoms and underlying pathology.
Main Methods:
- Transgenic mice with extra copies of the Plp1 gene were fed a curcumin-supplemented diet for 10 weeks.
- Phenotypical, histological, and immunohistochemical analyses were performed on the central nervous system.
- Control groups included Plp1 transgenic mice and wild-type mice on a normal diet.
Main Results:
- Curcumin improved motor performance by 50% and preserved myelinated axons by 35% in Plp1 transgenic mice.
- Curcumin reduced neuroinflammation, including astrocytosis, microgliosis, and lymphocyte infiltration.
- Curcumin restored glutathione levels, indicating a correction of oxidative imbalance in white matter.
Conclusions:
- Curcumin demonstrates potential as an antioxidant therapy for PMD.
- The findings suggest curcumin may be a viable treatment for PMD resulting from PLP1 gene duplication.
