AI Article Synopsis
- Prion diseases are brain disorders caused by misfolded proteins called prion proteins (PrP) that build up in the brain.
- Scientists have been trying to stop this misfolding, but a new drug strategy is being tested that aims to remove prions without affecting the prion proteins directly.
- A small molecule named compound 5 (ARN1468) has been found to help reduce prion levels in infected cells, showing promise for treating prion diseases even though it needs more testing to work with mice.
Article Abstract
Prion diseases are a group of neurodegenerative disorders characterized by the accumulation of misfolded prion protein (called PrP). Although conversion of the cellular prion protein (PrP) to PrP is still not completely understood, most of the therapies developed until now are based on blocking this process. Here, we propose a new drug strategy aimed at clearing prions without any direct interaction with neither PrP nor PrP. Starting from the recent discovery of SERPINA3/SerpinA3n upregulation during prion diseases, we have identified a small molecule, named compound 5 (ARN1468), inhibiting the function of these serpins and effectively reducing prion load in chronically infected cells. Although the low bioavailability of this compound does not allow studies in prion-infected mice, our strategy emerges as a novel and effective approach to the treatment of prion disease.
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Source |
|---|---|
| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC9289883 | PMC |
| http://dx.doi.org/10.1021/acs.jmedchem.2c00205 | DOI Listing |
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