“This is a promising alternative to conventional small molecule drugs which are often limited by poor penetration of the blood-brain barrier.”
And what is more surprising is it could be given to patients orally in a non-invasive manner or may be developed as a nasal spray.
Scientists have found a new way to stop the movement of mutant RNA and the production of toxic repeat proteins, which kill nerve cells in the most common types of motor neuron disease (MND) and frontotemporal dementia (FTD).
The new study, carried out by researchers at the University of Sheffield’s Institute of Translational Neuroscience (SITraN), also demonstrated that C9ORF72 nerve cells are more likely to survive when a peptide is used to prevent the transport of mutant repeated RNA molecules and the production of toxic repeat proteins, thereby preventing neurodegeneration.
The Sheffield researchers previously found that increased stickiness of a cell transporter known as SRSF1 causes improper transportation of rogue RNAs transcribed from the C9ORF72 gene, which is believed to be the most common cause of MND and FTD.
Instead of using traditional drugs, which don’t work well to stop the SRSF1 protein from sticking together, or invasive therapies to edit or change the activity of faulty genes, the new study found that a small peptide with a cell-penetrating module can stick to SRSF1 and stop the rogue repeat RNA from moving.
The peptide is made up of a brief chain of amino acids, or “bricks,” which are present in our body’s cells and tissues.
It’s interesting to note that the results, which were published in the journal Science Translational Medicine, also imply that the peptide may be administered to MND and FTD patients orally in a non-invasive way, such as by a nasal spray that could be created to penetrate the brain.
Potentially game-changing for the treatment of certain incurable neurodegenerative disorders, peptide-based inhibition of the harmful effects of enlarged repeat RNA and toxic repeat proteins might soon be a reality.
When they tested this “innovative approach by adding the peptide to the food eaten by fruit flies,” explains lead author Professor Guillaume Hautbergue, “not only did the peptides 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 indicates that the peptide is successfully halting the neurodegenerative condition’s development and aiding in the restoration of the function of the afflicted nerve cells.
The use of peptides to halt harmful mutations is a novel and promising therapy avenue that has not been investigated extensively until now.
MND and FTD are terrible diseases with no cure right now. This is a viable replacement for traditional small-molecule medications, which are often constrained by inadequate blood-brain barrier penetration.
FTD happens when nerve cells in the frontal and temporal lobes of the brain die. This makes the frontal and temporal lobes smaller. FTD usually affects people between the ages of 45 and 65, and it can change their behavior, personality, language, and the way they move. There is no way to stop or slow the progression of FTD, and there is no way to treat it.
MND is a debilitating disease that kills the cells that control movement. This means that people with MND can’t move, walk, talk, or breathe. There aren’t many ways to treat it, and there is no cure. After diagnosis, the majority of individuals with the condition are generally anticipated to survive two to five years. Donated skin tissue samples from people with Motor Neuron Disease were reprogrammed into nerve cells to conduct this research.
Within the next few years, it is hoped that innovative research will revolutionize clinical trials for the most prevalent forms of MND and FTD.
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