Thomas Gaj

This study uses CRISPR base editing to create HTT protein isoforms resistant to caspase-6 cleavage, reducing toxicity and neurodegeneration in a rodent HD model.

This study develops a CRISPR-Cas9 system targeting all HTT alleles in HD mouse models, reducing mutant HTT and improving disease symptoms.

This study develops a high-fidelity CRISPR-Cas13 system that reduces C9ORF72 repeat RNA expression in a rodent model, decreasing RNA foci and reversing transcriptional deficits without affecting normal C9ORF72 levels.

SPLICER improves exon skipping by editing both splice acceptor and donor sites, reducing aberrant splicing and enabling therapeutic applications in Alzheimer's disease.