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Accidental discovery leads to Parkinsonís disease cure in mice
www.studyfinds.org ^ | June 26, 2020 | by Brianna Sleezer

Posted on 06/29/2020 5:41:13 AM PDT by Red Badger

SAN DIEGO – Sometimes, scientific breakthroughs occur when researchers aren’t 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 Parkinson’s 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)

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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 Parkinson’s 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 Parkinson’s disease. In Parkinson’s, dopamine cells in the brain die. Thus, to assess their method, the researchers used a chemical that poisons dopamine neurons and creates symptoms of Parkinson’s 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 Parkinson’s 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 UCSD’s 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 Parkinson’s 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.

“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,” 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.


TOPICS: Business/Economy; Health/Medicine; History; Pets/Animals
KEYWORDS: alzheimer; astrocyte; disease; fibroblasts; helixmakemineadouble; huntingtons; mice; neurodegenerative; neurons; parkinsons; ptb
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1 posted on 06/29/2020 5:41:13 AM PDT by Red Badger
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To: Red Badger

Nature article:

https://www.nature.com/articles/d41586-020-01817-4


2 posted on 06/29/2020 5:46:06 AM PDT by Red Badger (To a liberal, 9-11 was 'illegal fireworks activity'..........................)
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To: Red Badger

This will please my mice.


3 posted on 06/29/2020 5:48:55 AM PDT by billorites (freepo ergo sum)
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To: Red Badger

Neurodegenerative Diseases:

Alzheimer’s disease (AD) and other dementias
Parkinson’s disease (PD) and PD-related disorders
Prion disease
Motor neurone diseases (MND)
Huntington’s disease (HD)
Spinocerebellar ataxia (SCA)
Spinal muscular atrophy (SMA)


4 posted on 06/29/2020 5:50:13 AM PDT by Red Badger (To a liberal, 9-11 was 'illegal fireworks activity'..........................)
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To: Red Badger

That’s quite amazing. Thanks for sharing.


5 posted on 06/29/2020 5:51:06 AM PDT by The Unknown Republican
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To: Red Badger

Don’t tell Fauci. This is clearly just anecdotal.....


6 posted on 06/29/2020 5:52:02 AM PDT by neodad (USS Vincennes (CG-49) Freedom's Fortress)
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To: neodad

Watson come here.


7 posted on 06/29/2020 5:55:56 AM PDT by DIRTYSECRET (urope. Why do they put up with this.)
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To: Red Badger

Excellent news! I have a personal stake in this.


8 posted on 06/29/2020 6:06:49 AM PDT by Blood of Tyrants (Where do you find the word "except" in the 2nd Amendment?)
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To: Blood of Tyrants

pass it around!..........................


9 posted on 06/29/2020 6:08:43 AM PDT by Red Badger (To a liberal, 9-11 was 'illegal fireworks activity'..........................)
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To: neodad

This is huge. Not only for Neurodegenerative diseases, but for spinal cord injuries as well!.................


10 posted on 06/29/2020 6:10:14 AM PDT by Red Badger (To a liberal, 9-11 was 'illegal fireworks activity'..........................)
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To: Blood of Tyrants
https://foldingathome.org/

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.

11 posted on 06/29/2020 6:15:45 AM PDT by texas booster (Join FreeRepublic's Folding@Home team (Team # 36120) Cure Alzheimer's!)
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To: Red Badger

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, Kennedy’s 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.


12 posted on 06/29/2020 6:16:32 AM PDT by Qiviut ("I have never wished death upon a man, but I have read many obituaries with pleasure" Mark Twain)
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To: texas booster

I was doing the FAH project several years ago. Do you know what team Freepers are on?


13 posted on 06/29/2020 6:19:12 AM PDT by Blood of Tyrants (Where do you find the word "except" in the 2nd Amendment?)
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To: Red Badger

Impossible! Everybody knows the only to cure these diseases is aborted fetal tissue. Just ask Michael J Fox. /s


14 posted on 06/29/2020 6:19:16 AM PDT by Shethink13
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To: Red Badger

I see an uptick in gliomas and hamartomas coming


15 posted on 06/29/2020 6:20:03 AM PDT by HangnJudge (When one is in a house of mirrors, everywhere you stand is ¬ďcenter)
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To: texas booster

I used google advanced and found the team number for FR. It’s 36120 if anyone wants to know.


16 posted on 06/29/2020 6:27:11 AM PDT by Blood of Tyrants (Where do you find the word "except" in the 2nd Amendment?)
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To: Blood of Tyrants
Yep, that's right.

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.

17 posted on 06/29/2020 6:56:34 AM PDT by texas booster (Join FreeRepublic's Folding@Home team (Team # 36120) Cure Alzheimer's!)
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To: Red Badger

Well, I guess if you’re a mouse and you have Parkinson’s... This is an awesome day for you.


18 posted on 06/29/2020 7:12:52 AM PDT by jerod (Nazi's were essentially Socialist in Hugo Boss uniforms... Get over it!)
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To: Red Badger

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.


19 posted on 06/29/2020 7:32:28 AM PDT by FLT-bird
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To: Red Badger

Neurodegenerative Diseases:

Progressive liberalism.


20 posted on 06/29/2020 7:43:38 AM PDT by glorgau
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