Brain Health

Putting A Gene Under “House Arrest” Curtails Two Devastating Brain Diseases

Putting A Gene Under “House Arrest” Curtails Two Devastating Brain Diseases about undefined
A “world leading” group of British scientists have created a groundbreaking approach to prevent the development of inherited forms of Lou Gehrig’s disease and frontotemporal dementia, a form of dementia that hit the headlines recently after Die Hard actor Bruce Willis was diagnosed with it. Although the research is still at an early stage, the scientists are optimistic a treatment can be developed in the form of a pill or nasal spray to bring these crippling conditions under control. Here’s the exciting news… Frontotemporal dementia (FTD) accounts for ten to 20 percent of dementia cases and is usually diagnosed in people ages 45 to 65. FTD was recently in the news because popular action star Bruce Willis has retired from making Hollywood movies following a diagnosis that, according to his family, apparently took years to come and has devastated his ability to work and live life normally. Bruce Willis is only 68-years-old. Lou Gehrig’s disease, more formally known as amyotrophic lateral sclerosis, is the most common type of motor neuron disease (MND). Researchers from the University of Sheffield’s Institute of Translational Neuroscience have discovered how to protect neurons from degeneration and prevent their death in genetic forms of these conditions.

What Causes FTD And MND?

Researchers are still trying to understand the causes of these two devastating neurological illnesses. However, research shows that FTD and MND can occur because of a mutation in a gene called C9orf72. The gene’s DNA provides instructions to RNA for making a protein that’s abundant in brain cells including cells that control movement (motor neurons). Healthy C9orf72 sends instructions to RNA for this to happen up to thirty times. But in a mutated gene the instruction is flawed, and the repeat instruction continues well beyond thirty. When mutant repeated RNA makes its journey from the cell nucleus to the cytoplasm of the cell, the protein it makes becomes toxic, causing cell degeneration and death. What the scientists discovered is a way to keep mutated RNA locked in the nucleus, blocking its transportation to the cytoplasm. The results are very promising…

Small Peptide Blocks Rogue RNA

The scientists achieved this in a novel way by using a peptide - a small assembly of amino acids or protein blocks - with a module that allows it to penetrate cells. The Sheffield team had already discovered the abnormal transportation of rogue RNA is caused by excessive stickiness of a cell transporter named SRSF1. Instead of using conventional drugs, which are inefficient in disrupting the stickiness of the SRSF1 protein, or invasive therapies to edit or modulate the activity of defective genes, they found the small peptide can stick to SRSF1 and effectively block the transportation of the rogue repeat RNA.

Works In The Laboratory

Injecting the peptide into the brains of mice carrying the mutation reduced expression of the toxic protein. It did the same by oral administration to fruit flies. Professor Guillaume Hautbergue, who led the study explains more: “When we tested our innovative approach by adding the peptide to the food eaten by fruit flies not only did the peptide block the damaging mutations which cause MND and FTD from being transported to the cell's nucleus, we actually saw an improvement in their neurofunction. “This means the peptide is effectively blocking the progression of the neurodegenerative condition and also helping to restore the function to the affected nerve cells… This concept of using peptides to block destructive mutations unlocks such an exciting and innovative treatment pathway which until now has not been explored by scientists.” He added, “MND and FTD are devastating diseases which currently have no cure. This is a promising alternative to conventional small molecule drugs which are often limited by poor penetration of the blood-brain barrier.”

Restoring Function To Damaged Cells Provides Hope For The Future

Dr. Brian Dickie, Director of Research at the MND Association, said, “These findings from a world leading research team in Sheffield demonstrate the importance of funding fundamental ‘discovery’ science. This work has provided important evidence in support of a completely new strategy to treat the most common inherited cause of both MND and FTD, with the ultimate goal of developing effective therapies for these devastating diseases.” Their unique strategy will have to be tested in humans of course. If all goes well the peptide could one day be given by mouth or in another non-invasive manner such as a nasal spray.

Our Takeaway

While the research is very exciting, it’s still in its infancy. Meanwhile, if you’re worried about any type of memory loss or neurological disorder, we encourage you to follow a memory-boosting, brain-saving lifestyle that can give you the best chance at side-stepping these illnesses. By combining regular exercise, a healthy diet rich in colorful vegetables, fresh fruit, lean meats and good fats together with stress management techniques and regular sleep, the research shows that you can positively affect other genes that are linked to the development of dementia and delay or even prevent memory loss. And let’s not forget the nutritional supplementation that we discuss regularly in this newsletter. Mother Nature has given us dozens of ways to protect the brain and sharpen memory and cognitive function, even in people who already have dementia symptoms. Stay hopeful. Best Regards, The Awakening From Alzheimer’s Team
https://www.sheffield.ac.uk/neuroscience-institute/news/scientists-discover-how-prevent-death-nerve-cells-most-common-genetic-forms-mnd-and-dementia

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