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The knockdown of SAMMSON disrupts mitochondrial functions by targeting downstream p32 and can thereby increase the efficacy of MAPK inhibition-targeted therapy 69 (Fig

In antisense oligonucleotide (ASO) therapies, a C9orf72 sense transcript-targeting antisense oligonucleotide BIIB078 exhibited robust efficacy in the preclinical mouse and cellular models (Donnelly et al., 2013

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