Scientists find the key to new Parkinson’s disease treatments

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Parkinson’s disease is a brain disorder that affects movement and worsens over time. One of its main causes is the buildup of a harmful protein called α-synuclein inside brain cells.

These protein clumps damage nerve cells and lead to problems such as shaking, stiffness, and trouble with balance or walking. But scientists are still trying to fully understand why this damage happens and how it could be stopped.

Now, researchers from KAIST in South Korea, working with teams at University College London and the Francis Crick Institute, have discovered something new: a natural process called RNA editing may play a major role in how Parkinson’s disease develops. Their study, published in Science Advances, could open the door to a completely new kind of treatment.

The team, led by Professor Minee L. Choi, focused on cells in the brain called astrocytes. These cells don’t carry signals like neurons do, but they support and protect neurons and help manage immune responses in the brain.

To investigate how these cells react in Parkinson’s disease, the scientists created a lab model using stem cells from patients with the condition. They grew astrocytes and neurons from these stem cells and then exposed them to clumps of α-synuclein—similar to what happens in the brains of real patients.

What they found was surprising. When these toxic protein clumps were introduced, they triggered a strong immune reaction in the astrocytes. Specifically, early-stage α-synuclein clumps (called oligomers) activated danger-detection systems in the brain, such as the Toll-like receptor and interferon pathways.

These are usually used to fight off infections. But instead of helping, this strong immune reaction led to inflammation, which made things worse for nearby neurons.

Digging deeper, the team discovered that an enzyme called ADAR1 was involved. This enzyme normally edits RNA—the genetic instructions that tell cells what to do—by changing one of the building blocks in RNA (adenosine) into another (inosine). This process, called A-to-I RNA editing, usually helps control the immune system and prevent overreactions.

However, in this case, ADAR1 was behaving differently. When the astrocytes were exposed to α-synuclein clumps, ADAR1 was altered. It produced a slightly different form of the protein that edited RNA in an abnormal way. Instead of helping to calm the immune system, it made it worse by focusing on genes that promote inflammation.

Even more importantly, the researchers saw the same pattern in actual brain tissue from people who had died from Parkinson’s disease—not just in the lab models. This confirms that RNA editing gone wrong is not just a lab observation, but something that really happens in the brains of patients.

This is the first time scientists have directly linked faulty RNA editing to the inflammation that damages neurons in Parkinson’s disease. It’s a big step forward in understanding the disease. Professor Choi said that this discovery points to a new layer of disease control—one that happens after DNA is read, but before proteins are made.

What makes this research especially exciting is its potential for future treatments. Right now, Parkinson’s therapies mostly try to replace lost brain chemicals or reduce symptoms.

This study suggests that targeting RNA editing—specifically, the ADAR1 enzyme—could offer a completely different way to fight the disease at its root by controlling inflammation before it harms brain cells.

In conclusion, this study shows that RNA editing is deeply involved in the brain’s response to the toxic protein clumps that cause Parkinson’s disease. If scientists can find a way to correct or block the abnormal editing process, they might be able to prevent the damaging inflammation and slow or even stop the disease.

This new insight could lead to the next generation of therapies for Parkinson’s disease and other brain disorders involving inflammation.

If you care about Parkinson’s disease, please read studies that Vitamin B may slow down cognitive decline, and Mediterranean diet could help lower risk of Parkinson’s.

For more health information, please see recent studies about how wheat gluten might be influencing our brain health, and Olive oil: a daily dose for better brain health..

The research findings can be found in Science Advances.

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