Dual-targeting CRISPR-CasRx reduces C9orf72 ALS/FTD sense and antisense repeat RNAs in vitro and in vivo
Liam Kempthorne, Deniz Vaizoglu, Alexander J. Cammack, Mireia Carcolé, Martha J. Roberts, Alla Mikheenko, Alessia Fisher, Pacharaporn Suklai, Bhavana Muralidharan, François Kroll, Thomas G. Moens, Lidia Yshii, Stijn Verschoren, Benedikt V. Hölbling, Francisco C. Moreira, , Adrian M. Isaacs
This study demonstrates that CRISPR-CasRx reduces C9orf72 repeat RNAs and DPRs in vitro and in vivo, suggesting its therapeutic potential for ALS/FTD.
Nature Communications · Jan 8, 2025Peer-reviewedNeuroscience