Treating Parkinson's Disease With A Single Shot

in StemSocial4 years ago

The deletion of a single gene changes regular cells into neurons allowing for fixing a damaged brain. This allowed scientists to cure lab mice of blindness or Parkinson's with a single shot.

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Image by Angelo Esslinger from Pixabay

Neurodegenerative diseases such as Parkinson's or Alzheimer's disease are a modern-day scourge. As the average life-span gets longer the modern seniors who suffer from these diseases much more often we get. Nowadays, we have roughly 50 million people who suffer from dementia caused by brain damage.

Every year, 10 million people are diagnosed with this type of dementia. We are expecting the total number of people with this disease to increase to 82 million by 2030 and 150 million by 2050. And it is estimated that up to 80 % of dementia cases are connected to Alzheimer's disease.

Neurodegenerative diseases are caused by neurons in the brain dying. While new neurons are being created in the brain throughout the whole life the regenerative properties of the brain are minimal and thus the damage outpaces the healing over time. This is why scientists all over the world are working on new treatments that could return healthy neurons into the brain.

When it comes to Parkinson's disease it is caused by the substantia nigra neurons dying. These neurons produce dopamine. After their death, the brain suffers from a lack of this very important substance leading into the wide range of problems associated with Parkinson's.

To a certain degree, we can mitigate the symptoms of Parkinson's by drugs. But as the diseases progress the doses need to be increased and the doses eventually reach such a high level that they cannot be increased any further.

Deleting a gene => New neurons

Some modern treatment methods bring some relief to patients – for example, deep brain stimulation using electrodes directly on the relevant brain centers. But, this is quite the extensive surgery that cannot be performed on all patients. That is why scientists are searching for new, less dangerous, and more effective treatments.

Two completely independent teams seem to have succeeded. The first was led by Xian-Dong Fu from the University of California in San Diego published their study in the journal Nature. And a Chinese team led by Hui Yang from the Shanghai Institute for Biological Sciences of the Chinese Academy of Sciences published their work in the journal Cell.

Both the teams discovered a simple way of transforming common types of cells into neurons. All they need to do is to block a single gene from working. More precisely the PTBP1 gene. Even simple skin cells called fibroblasts can undergo this metamorphosis in laboratory conditions. But getting their to transform inside a living being is not that easy. Yet, both the American and Chinese teams managed to do it.

The scientists used lab mice in which the neurons are forcefully degenerating and dying so they suffer from similar symptoms as the human patients with Parkinson's. Then they put a genetically modified but harmless virus that destroys the RNA made based on the instructions from the PTBP1 gene.

The gene itself is kept unharmed but the instruction for creating the protein which is synthesized based on the gene is suppressed. And cells that do not have this protein transform into neurons.

Treats Parkinson's and Blindess

The American team managed to achieve the creation of new neurons that produce dopamine in the brains of mice suffering from Parkinson's disease. The symptoms of the diseases – especially coordination problems – drastically decreased in just a few months. The Chinese team tried out the creation of new neurons by blocking the PTBP1 gene not only in the brain but in the eye as well.

The mice suffering from Parkinson's also started producing dopamine again in the brains and the symptoms were reduced as well. Then Yang and his coworkers tested the new treatment on mice suffering from blindness as a result of neurons dying in the retina.

These cells called ganglion cells to get signals from light-sensitive cells in the retina and convey them into the eye nerves which carries them into the brain. The death of the ganglion cells results in a break in the link leading to blindness.

But there are so-called Müller glial cells in the retina which have a support role in the retina. The human retina has around 10 million of them. Transforming part of them into neurons capable of doing the job of ganglion cells led to the mice getting their vision back.

If you are looking forward to having your or your close one's Parkinson's or blindness treated – well, you will have to wait. There is still a long road ahead of us before these treatments are tested enough to be used on human patients.

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 4 years ago  

One thing about degenerative disease or most Brain diseases is that , they mostly managed and difficult to fully treat.

As we all know, brain cells are not regenerative and once a damage done, recovery is seemingly impossible