G2C4 targeting antisense oligonucleotides potently mitigate TDP-43 dysfunction in C9orf72 ALS/FTD human neurons
Jeffrey D. Rothstein, Victoria Baskerville, Sampath Rapuri, Emma Mehlhop, Paymaan Jafar‐Nejad, Frank Rigo, Frank Bennett, Sarah Mizielinska, Adrian M. Isaacs, Alyssa N. Coyne
This study shows that targeting G2C4 antisense repeat RNA, not G4C2 sense RNA, mitigates TDP-43 dysfunction in C9orf72 ALS/FTD patient neurons, explaining the failure of previous G4C2 ASO clinical trials.
bioRxiv · Jun 26, 2023 · PreprintNeuroscience