NeurosciencebioRxivPreprint
Protein-stabilizing and neurotransmission-potentiating activities of a synaptic chaperone modify spinal muscular atrophy in model mice
Yoon-Ra Her, Andrea Fuentes, Rashmi Kothary…·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.
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