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San Diego School of Medicine scientists found a way to activate and deactivate PTB gene in mice, thereby eliminating Parkinson’s disease through genetic changes.
While attempting to determine the role of protein in connective tissue cells, San Diego School of Medicine, scientists found a way to transform multiple types of cells into neurons. The PTB protein that was being researched has the property of activating and deactivating genes within a cell.
To better understand its functionality, the researchers deactivated the PTB gene in fibroblasts, which are a type of connective tissue cell, with a technique called siRNA.
The fibroblasts were grown in a petri dish, and the PTB gene in them was switched off. The scientists waited for two weeks to see any changes and developments in the genes.
The scientists on observation discovered that after about a fortnight, only a few fibroblasts were left, and the rest had transformed into neurons. Further experiments with other cells also gave the same results.
The scientists silenced the PTB gene in brain cells known as astrocytes and were able to generate neurons.
Professor Xiang-Dong Fu, lead author of the San Diego School of Medicine study, said: “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.
“The fact that we could produce so many neurons in such a relatively easy way came as a big surprise.”
Subsequent experiments made the researchers realize that they could also turn other cells into neurons.
This encouraged the researchers to try and replicate the experiment to generate new neurons from cells for neurodegenerative diseases like Parkinson’s.
In Parkinson’s disease, the dopamine cells die out. The San Diego School of Medicine researchers used a chemical to kill dopamine cells in mice to create Parkinson like symptoms. Here they replicated the earlier experiment and switched off the PTB gene in the astrocyte cells with siRNA method. After a period of time, 30 percent of the astrocytes turned into neurons that produce the neurotransmitter dopamine, which were able to function normally and send signals to the brain. The mice were able to regain some of their motor functions.
Moreover, it was found that the recovered functionality of the neurons was permanent and the mice did not revert back to any dysfunction for the rest of their life.
Dr. William Mobley, Professor of Neurosciences at UCSD’s School of Medicine and study co-author, said: “I was stunned at what I saw.
“This whole new strategy for treating neurodegeneration gives hope that it may be possible to help even those with advanced disease.”
The experiment has great potential for treating degenerative neural disorders, but the research is still in the experimental stage and, to date, has been successful on animals. The method has to be more rigorously tested. The team plans to optimize their methods and test the approach in mouse models that mimic Parkinson’s disease through genetic changes.
The researchers have also patented their treatment in hopes of being able to move forward with human testing at a later date.
Dr. Fu said in his press statement: “It’s my dream to see this through to clinical trials, to test this approach as a treatment for Parkinson’s disease, but also many other diseases where neurons are lost, such as Alzheimer’s and Huntington’s diseases and stroke.
“And 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?”
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