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Peptide-Based Therapy for ALS Examined in Lab Testing

A potential new peptide-based therapy for a certain form of amyotrophic lateral sclerosis (ALS) is reported in Science Translational Medicine this week. Hexanucleotide repeat expansions in the C9ORF72 gene are the most common genetic cause of familial ALS and frontotemporal dementia (FTD), and research has shown that the hexanucleotide expansions cause the noncanonical translation of C9ORF72 transcripts into neurotoxic dipeptide repeat proteins (DRPs) that contribute to the conditions' hallmark neurodegeneration. Aiming to block the nuclear export of these pathological transcripts as a therapeutic strategy, a team led by scientists from the University of Sheffield developed a cell-penetrant peptide that could prevent the nuclear exit of pathological C9ORF72 RNA transcripts, demonstrating that it inhibits the production of DPRs in rat and human cell models. The peptide was also shown to block neurotoxic DPR production in flies and mice in vivo, as well as ameliorate locomotor deficits in a fruit fly model of ALS/FTD. "These findings demonstrate that disrupting the production of DPRs in cellular and animal models of ALS/FTD might be a strategy to ameliorate neurodegeneration in these diseases," the study's authors conclude.