Scientists have found a novel way to block the transportation of mutant RNA and subsequent production of toxic repeat proteins which lead to the death of nerve cells in the most common genetic subtypes of motor neurone disease (MND) and frontotemporal dementia (FTD).

The new study, conducted by researchers at the University of Sheffield’s Institute of Translational Neuroscience (SITraN), also showed that using a peptide to stop the transport of mutant repeated RNA molecules and production of toxic repeat proteins actually increases the survival of C9ORF72 nerve cells - protecting them against neurodegeneration.

The Sheffield team previously discovered the abnormal transportation of the rogue RNAs copied from the C9ORF72 gene - known to be the most frequent cause of MND and FTD - 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, the new study found that a small peptide incorporating a cell-penetrating module can stick to SRSF1 and effectively block the transportation of the rogue repeat RNA.

https://www.science.org/doi/10.1126/scitranslmed.abo3823

As someone for whom ALS runs in my family, I really hope this leads to more successful trials and eventually an effective treatment on the market.

In fruit flies, not humans

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Why do articles repeat the same thing 3 times in a row with very slightly different wording all the time? In the sub title intro statement, in the bullet text of an image, and then again as the intro to the article where they FINALLY explain the acronyms that they should have used as the one and only explanation of it in the title description. I don't need to read the same thing 3 times in a row to get through the intro of an article, it is super annoying and feels like a school child trying to hit 500 words on their essay.