A team of scientists from the University of California in San Diego has been conducting a study about Parkinson's disease affecting nerve cells, or neurons. They were 'stunned' that their mice model were cured of the disease during their experiments.
Parkinsons's disease is characterized by neurons unable to produce substantial amounts of dopamine to regulate movement. This results in tremors, slow movement, imbalance, and stiffness.
The researchers had been developing various treatments for the disease, including physiotherapy for patients and drug developments with their mouse model.
One particular protein they focused on was polypyrimidine tract binding (PTB), which turns genes 'on or off' in cells. As they sought to understand how PTB affects cells, they 'silenced' the protein as they grew connective tissue cell fibroblasts in their lab.
After some time, with the PTB protein silenced, fibroblasts became neurons. More recently, they saw that astrocytes, a type of brain cell, also became neurons with silenced PTB proteins.
Cured Neurons
'Researchers around the world have tried many ways to generate neurons in the lab, using stem cells and other means, so we can study them better, as well as to use them to replace lost neurons in neurodegenerative diseases,' said Dr. Xiang-Dong Fu. It was a 'big surprise' that they could produce numerous neurons easily.
In their mouse model, they attempted to apply the same principles with Parkinson's. Silencing PTB in the rodents caused 30% of their astrocytes to turn into normal-growing neurons, curing the lack of dopamine. The single treatment completely restored normal movement.
Dr. William Mobley said, 'I was stunned at what I saw.' Treating neurodegeneration gave the team hope with expanding the research to other advanced diseases.
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Treating Other Diseases
Professor David Dexter from Parkinson's UK explained, 'Cell transplants have, for a long time, aimed to replace lost cells in Parkinson's, but their effectiveness has been limited since they struggle to integrate and function effectively within the brain. This new technique has overcome this major hurdle in mice and opens the door to an exciting new treatment approach, which may be able to reverse Parkinson's in people, in the future."
Even with the promising result, there is still plenty of testing to be done before human trials can begin. 'Advances in technologies like this are vital and this is promising and well-conducted early-stage research,' said Dr. Sara Imarisio from Alzheimer's Research UK, yet moving from mice models to human trials is still far off. The team will also need to consider the possible adverse effects of this single treatment.
Dr. Fu expressed his dream to one day see the treatment's clinical trials and even explaining it to other central nervous system diseases such as Alzheimer's and Huntington's. 'Dreaming even bigger, what if we could target PTB to correct defects in other parts of the brain, to treat things like inherited brain defects?' said Fu. Parkinson's UK continues to raise support for research to 'end the fear, heartbreak, and harm that diseases like Parkinson's cause," said Dr. Imarisio.
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