RNA-Targeting CRISPR/Cas13d System Eliminates Disease-Related Phenotypes in Pre-clinical Models of Huntington's Disease.
Kathryn H. Morelli, Qian Wu, Maya L. Gosztyla, Hongshuai Liu, Chuangchuang Zhang, Jiaxu Chen, Ryan J. Marina, Kari S. Lee, Krysten L. Jones, Wenzhen Duan, Gene Wei-Ming Yeo
This study develops a CRISPR-Cas13d system targeting CAG-expanded HTT RNA in HD models, reducing mutant mRNA and reversing disease phenotypes without significant off-target effects.
bioRxiv · Jan 23, 2022 · PreprintNeuroscience