Article

Pharmacologic eIF2B Activation Rescues Neuropathy in CMT2 Subtypes by Normalizing the Integrated Stress Response

2026-08-06

Abstract excerpt

Among the many subtypes of Charcot–Marie–Tooth (CMT) disease, several result from mutations in genes encoding aminoacyl-tRNA synthetases, enzymes required for tRNA charging during cytoplasmic and mitochondrial translation. We report that activation of the integrated stress response (ISR) pathway is a shared molecular feature of tRNA synthetase-associated and other axonal CMT subtypes. RTX-117, a CNS-penetrant smal...

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Literature Corpus work: 4e59a286-595c-51e1-9040-3caa35b66760
DOI: 10.64898/2026.08.05.743063

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