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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.

The Scan

ChatGPT Does As Well As Humans Answering Genetics Questions, Study Finds

Researchers in the European Journal of Human Genetics had ChatGPT answer genetics-related questions, finding it was about 68 percent accurate, but sometimes gave different answers to the same question.

Sequencing Analysis Examines Gene Regulatory Networks of Honeybee Soldier, Forager Brains

Researchers in Nature Ecology & Evolution find gene regulatory network differences between soldiers and foragers, suggesting bees can take on either role.

Analysis of Ashkenazi Jewish Cohort Uncovers New Genetic Loci Linked to Alzheimer's Disease

The study in Alzheimer's & Dementia highlighted known genes, but also novel ones with biological ties to Alzheimer's disease.

Tara Pacific Expedition Project Team Finds High Diversity Within Coral Reef Microbiome

In papers appearing in Nature Communications and elsewhere, the team reports on findings from the two-year excursion examining coral reefs.