Yu Tang

This study evaluates allele-specific targeting of the mutant TOR1A{Delta}E allele using NmCas9, showing high specificity and efficacy in reducing ubiquitin accumulation in DYT1 fibroblasts.

This study uses human neurons to model DYT1 dystonia, revealing LMNB1 dysregulation as a key factor in disease pathology.

AsCRISPR is a web tool for designing allele-specific gRNAs to target mutant alleles or SNPs while preserving normal alleles, with evaluations of on-target efficiency and off-target potential.