Protein-stabilizing and neurotransmission-potentiating activities of a synaptic chaperone modify spinal muscular atrophy in model mice
Yoon-Ra Her, Andrea Fuentes, Rashmi Kothary, Lucı́a Tabares, Umrao R. Monani
This study identifies an Hspa8G470R variant that suppresses SMA in mice by increasing SMN levels and enhancing neuromuscular transmission, suggesting new therapeutic targets.
bioRxiv · Feb 24, 2026 · PreprintNeuroscience