(@lealescouzeres.bsky.social)

🎉 Excited to share our latest preprint📰 ! www.biorxiv.org/cgi/content/... Many thanks to everyone involved in this project ! Using a zebrafish model 🐟 and iPSC-derived motor neurons 🔬 we show that restoring the calpastatin/calpain pathway preserves motoneuron survival and function in C9orf72 ALS!

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Therapeutic modulation of the calpastatin/calpain pathway restores calpain-mediated synaptic proteolysis and preserves motor neurons survival and function in C9orf72 ALS A hexanucleotide repeat expansion (GGGGCC) in the C9orf72 gene is the most prevalent genetic cause of ALS, with early neuromuscular junction (NMJ) dysfunction being a key pathological feature. Current... https://www.biorxiv.org/cgi/content/short/2025.12.08.692907v1