Dual-targeting CRISPR-CasRx reduces C9orf72 ALS/FTD sense and antisense repeat RNAs in vitro and in vivo

Dual-targeting CRISPR-CasRx reduces C9orf72 ALS/FTD sense and antisense repeat RNAs in vitro and in vivo

Abstract The most common genetic cause of frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS) is an intronic G4C2 repeat expansion in C9orf72. The repeats undergo bidirectional transcription to produce sense and antisense repeat RNA species, which are translated into dipeptide repe...

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Main Authors: 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, Eszter Katona, Rachel Coneys, Paula de Oliveira, Yong-Jie Zhang, Karen Jansen, Lillian M. Daughrity, Alexander McGown, Tennore M. Ramesh, Ludo Van Den Bosch, Gabriele Lignani, Ahad A. Rahim, Alyssa N. Coyne, Leonard Petrucelli, Jason Rihel, Adrian M. Isaacs
Format: Article
Language:English
Published: Nature Portfolio 2025-01-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-024-55550-x
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