Skip to comments.Accidental discovery leads to Parkinsonís disease cure in mice
Posted on 06/29/2020 5:41:13 AM PDT by Red Badger
SAN DIEGO Sometimes, scientific breakthroughs occur when researchers arent exactly looking for them. While attempting to better understand the function of a protein in connective tissue cells, UC San Diego School of Medicine scientists found a way to transform multiple types of cells into neurons. This discovery has led to the development of a treatment that eliminates symptoms of Parkinsons disease in mice.
The protein researchers were studying, called PTB, is known for its general role in activating or deactivating genes within a cell. In an attempt to better understand how PTB contributes to cell function, researchers silenced the PTB gene using a technique called siRNA in a type of connective tissue cell, known as a fibroblast. The researchers grew the fibroblasts in petri dishes, silenced PTB, and waited a couple of weeks to check on the fibroblasts and observe any changes.
Left: mouse astrocytes (green) before reprogramming; Right: neurons (red) induced from mouse astrocytes after reprogramming with PTB antisense oligonucleotide treatment. (Image credit: UC San Diego Health Sciences)
When the researchers checked the fibroblasts, they were shocked. They found that very few fibroblasts remained in the dishes. Instead, the dishes contained mostly neurons. Unintentionally, they had discovered a way to turn fibroblasts into neurons.
In subsequent experiments, the researchers found that they could also turn other cells into neurons. When they silenced PTB in a type of non-neuronal brain cell known as an astrocyte, they were also able to generate 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, says lead author of the study, Xiang-Dong Fu, in a release. The fact that we could produce so many neurons in such a relatively easy way came as a big surprise.
Fu is a Distinguished Professor in the Department of Cellular and Molecular Medicine at UC San Diego School of Medicine. Scientists stunned after Parkinsons disease test
Once researchers realized that they could easily create neurons from other types of cells, they decided to see if the method could be used to treat neurodegenerative diseases, such as Parkinsons disease. In Parkinsons, dopamine cells in the brain die. Thus, to assess their method, the researchers used a chemical that poisons dopamine neurons and creates symptoms of Parkinsons disease in mice.
After using the chemical to kill dopamine neurons in the mice, researchers silenced PTB. They found that 30% of astrocytes turned into neurons. Moreover, these newly generated neurons seemed to grow normally and even began to send connections to other parts of the brain, like normal neurons.
When the researchers looked at mouse behavior, they also found that silencing PTB completely restored movement function. Moreover, even though the PTB-silencing treatment was only administered once, the mice did not show any symptoms of Parkinsons disease for the remainder of their lifetime.
I was stunned at what I saw, said study co-author Dr. William Mobley, Distinguished Professor of Neurosciences at UCSDs School of Medicine. This whole new strategy for treating neurodegeneration gives hope that it may be possible to help even those with advanced disease. Not quite ready for humans
While the treatment is promising for the treatment of neurodegenerative diseases in humans, it still needs to go through more rigorous testing before being ready for human trials. In the next steps, the researchers plan to test the method with other mouse models of Parkinsons disease. These models involve genetic changes rather than chemical destruction of dopamine neurons.
The researchers have also patented their treatment in hopes of moving it forward to testing in humans.
Its my dream to see this through to clinical trials, to test this approach as a treatment for Parkinsons disease, but also many other diseases where neurons are lost, such as Alzheimers and Huntingtons diseases and stroke, Fu says. 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?
I intend to spend the rest of my career answering these questions, he says.
The study is published in Nature.
This will please my mice.
Alzheimers disease (AD) and other dementias
Parkinsons disease (PD) and PD-related disorders
Motor neurone diseases (MND)
Huntingtons disease (HD)
Spinocerebellar ataxia (SCA)
Spinal muscular atrophy (SMA)
That’s quite amazing. Thanks for sharing.
Dont tell Fauci. This is clearly just anecdotal.....
Watson come here.
Excellent news! I have a personal stake in this.
pass it around!..........................
This is huge. Not only for Neurodegenerative diseases, but for spinal cord injuries as well!.................
We all have a personal stake in this research - whether we know it today, or not.
This is one research group that has been focusing on a branch of disease research called misfolding proteins.
FReepers have been involved since the project started in the early 2000's and have been folding continuously since.
Motor neurone diseases (MND)
The motor neuron diseases (MNDs) are a group of progressive neurological disorders that destroy motor neurons, the cells that control skeletal muscle activity such as walking, breathing, speaking, and swallowing. This group includes diseases such as amyotrophic lateral sclerosis, progressive bulbar palsy, primary lateral sclerosis, progressive muscular atrophy, spinal muscular atrophy, Kennedys disease, and post-polio syndrome.
ALS - wouldn’t it be something else (words fail me here) if the death sentence of ALS was no longer a death sentence? I know folks who have died from ALS & also know folks currently coping with Parkinsons ... just had an old gentleman I’ve known for decades, die after years of being bedridden with Parkinsons.
It’s just “mice” at this point, but I wish them Godspeed with their research & hope it tanslates to humans with the same wonderful results.
I was doing the FAH project several years ago. Do you know what team Freepers are on?
Impossible! Everybody knows the only to cure these diseases is aborted fetal tissue. Just ask Michael J Fox. /s
I see an uptick in gliomas and hamartomas coming
I used google advanced and found the team number for FR. It’s 36120 if anyone wants to know.
I have also kept it as my tagline for ~20 years ...
Thanks for rejoining the process. It is the same concept as before, but with GPUs doing most of the heavy lifting the computational speeds are amazing compared to the CPU only folding that most of us used to do.
Well, I guess if you’re a mouse and you have Parkinson’s... This is an awesome day for you.
I hope this works. My mom died from Parkinson’s disease. I’d love to add it to the list of diseases conquered by medical science.
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