Repairing the nuclear pore in ALS

Abstract

Biomedicine Most cases of amyotrophic lateral sclerosis (ALS) are of unknown origin (sporadic ALS). The nuclear pore complex is known to be involved in familial ALS; however, whether and how it also plays a role in sporadic ALS remain to be elucidated. Coyne et al. used human tissue and induced pluripotent stem cell–derived motor neurons from patients with familial and sporadic ALS to show that the protein CHMP7, previously shown to be involved in nuclear pore complex homeostasis in yeast, was increased in both ALS subtypes. An antisense oligonucleotide targeting CHMP7 reduced cellular abnormalities and promoted neuronal survival, suggesting that CHMP7 is a potential target for treating both sporadic and familial ALS. Sci. Transl. Med. 13 , eabe1923 (2021).