G2C4 targeting antisense oligonucleotides potently mitigate TDP-43 dysfunction in human C9orf72 ALS/FTD induced pluripotent stem cell derived neurons
G2C4 targeting antisense oligonucleotides potently mitigate TDP-43 dysfunction in human C9orf72 ALS/FTD induced pluripotent stem cell derived neurons
Jeffrey D. Rothstein,Victoria Baskerville,7 Authors,Alyssa N. Coyne
2023 · DOI: 10.1007/s00401-023-02652-3
Acta Neuropathologica · 14 Citations
TLDR
It is shown that G2C4 antisense, but not G4C2 sense, repeat RNA is sufficient to induce TDP-43 dysfunction in induced pluripotent stem cell (iPSC) derived neurons ( iPSNs) and insights are provided into a possible explanation for the recent G 4C2 ASO clinical trial failure.