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.

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