Wagamaga OP t1_jct2bt9 wrote
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.
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