August 27, 2020 by Dean Bellefeuille

Researchers at the UC San Diego School of Medicine who thought they were examining connective tissue cell protein got quite a shock when they instead stumbled upon a treatment to eradicate the observable symptoms of Parkinson’s disease in mice. Not only that, but the treatment could potentially be useful in many different conditions that call for the need to replace damaged tissue: diabetes, spinal cord injuries, even coronary disease.

The study centered on an siRNA technique, in which researchers grew fibroblasts (a type of connective tissue cell) in petri dishes while silencing the protein PTB. After several weeks, they were surprised to learn the majority of fibroblasts had changed into neurons.

Xiang-Dong Fu, lead researcher of the study, stated, “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.”

The research team then decided to test their new theory on Parkinson’s disease in mice, finding that it completely restored movement and functionality for the remainder of the mice’s lifetime – even those with an advanced case of the disease.

Needless to say, the method to determine if these findings transfer to humans takes some time and extreme care to make certain of safety; nonetheless, for those having difficulties with the disease and other neurodegenerative conditions, it’s cause for optimism.

As Fu explains, “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 disease 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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