This work is the result of a fantastic collaboration over the past few years in Montreal between the Patten lab at @inrsciences.bsky.social , The Neuro's EDDU (Early Drug Discovery Unit) @theneuro.bsky.social , and the group of Antoine Dufour @ucalgary.bsky.social 🧬

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...

🎉 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!