NeurosciencebioRxivPreprint
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…·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.
3 citations