CRISPR-Cas base editing altered HTT RNA splicing and reduced toxic huntingtin fragments in a mouse model of Huntington’s disease. Treated mice showed...
A six-year-old girl died after receiving an experimental CRISPR-Cas base-editing treatment for a nonfatal neurodevelopmental disorder in China. The...
Spatial Perturb-seq enables pooled in vivo CRISPR screening with single-cell, whole-transcriptome readouts preserved within intact tissue...
Researchers in the US report a potentially new therapeutic strategy for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Using...
Researchers have developed a compact, all-in-one CRISPR-dCas9-based repressor platform delivered via AAV, effectively silencing gene expression...
