Article

G <sub>2</sub> C <sub>4</sub> targeting antisense oligonucleotides potently mitigate TDP-43 dysfunction in C9orf72 ALS/FTD human neurons

2023-06-26

Abstract excerpt

The G 4 C 2 repeat expansion in the C9orf72 gene is the most common genetic cause of Amyotrophic Lateral Sclerosis and Frontotemporal Dementia. Many studies suggest that dipeptide repeat proteins produced from this repeat are toxic, yet, the contribution of repeat RNA toxicity is under investigated and even less is known regarding the pathogenicity of antisense repeat RNA. Recently, two clinical trials targeting...

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Literature Corpus work: 3f164b53-8ff5-52fd-a3a1-6421030742a7
DOI: 10.1101/2023.06.26.546581

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