NeuroscienceNature CommunicationsPeer-reviewed
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…·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.