A groundbreaking study led by Yulan Xiong, an assistant professor of neuroscience at UConn Health, has identified a potential treatment for Parkinson’s disease.
Most familial cases of Parkinson’s are caused by mutations in a gene called LRRK2, which codes for a protein known as daradarin.
The mutations lead to an overproduction of this protein, something that, until now, scientists didn’t know how to control.
How It Works
Xiong and her lab performed a genome-wide screening to identify potential regulators of LRRK2.
They discovered that an enzyme called ATIC regulates LRRK2 at the mRNA level, providing the cell with instructions to build the protein.
This is a departure from the previous understanding, which focused on LRRK2’s enzymatic activity.
The Compound and its Mechanism
They found that AICAr, the precursor of ATIC substrate, can significantly reduce LRRK2 levels. ATIC substrate brings in a binding protein called AUF-1 to specific regions of LRRK2 mRNA.
AUF-1, in turn, recruits another enzyme complex, DCP1/2, together reducing the LRRK2 levels.
AICAr shows promise in treating metabolic and cardiovascular disorders but currently cannot pass the blood-brain barrier, which is essential for treating Parkinson’s disease.
Xiong and her collaborators are working on modifying AICAr to overcome this limitation.
“This is the first study to find out the mechanism. It’s also important that we identified the compound that can directly decrease LRRK2 levels, which means that we can use this compound to treat Parkinson’s patients,” says Xiong.
Future Prospects
Xiong and her lab are planning further animal trials and are hopeful about moving to human clinical trials soon. They are also working with UConn’s Technology Commercialization Services (TCS) to protect and commercialize this innovative discovery.
TCS has already filed a non-provisional patent application for the technology and is facilitating connections between Xiong and companies specializing in Parkinson’s disease treatment.
This discovery opens new doors in understanding and treating Parkinson’s disease, offering hope to millions suffering from this debilitating condition.
If you care about Parkinson’s disease, please read studies about Vitamin E that may help prevent Parkinson’s disease, and Vitamin D could benefit people with Parkinson’s disease.
For more information about brain health, please see recent studies about new way to treat Parkinson’s disease, and results showing COVID-19 may be linked to Parkinson’s disease.
The study was published in The EMBO Journal.
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