The Technology
Parkinson’s disease is a very common neurodegenerative disease. It affects at least 1% of the population above the age of 65. Although there is palliative treatment for the disease’s motor symptoms, there is no treatment to prevent or decrease the wide range of symptoms caused by this devastating condition. One of the reasons for the failure of finding the proper treatment for the disease is that there is still no compound that can efficiently decrease the levels and aggregation of a-synuclein, which is the main player in the pathogenesis of the disease. Even all clinical trials of antibodies against a-synuclein failed to improve the disease. The research lab recently developed a cell-penetrating peptide that prevents a-synuclein SUMOylation and enhances its proteasomal degradation. By applying this peptide to primary neuronal cultures, we observe that low micromolar concentrations strikingly reduce the levels and pathology of α-synuclein in neurons. It also decreases endogenous levels of a-synuclein in mouse brains when administered intraperitoneally, suggesting that it enters the CNS and exerts a robust effect. Most importantly, the peptide reduces a-synuclein pathology of the PD mouse model of a-synuclein pre-formed fibrils (a-SynPFF). The decrease of both the levels and aggregation α-synuclein in neurons and in vivo suggest that this peptide may be able not only to prevent but also to rescue from the neurodegeneration observed in Parkinson’s disease and other α-synucleinopathies.
Advantages
- Novel mechanism of action
- Decoy-peptide based strategy
- Decreasing both concentration and aggregation of a-synuclein
Applications and opportunities
- Treating Parkinson’s disease and other alpha-synucleinopathies
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