CRISPR in Gene Therapy; Safety Issues
January 3, 2023 | Terry Sharrer
“The treatment, which was designated CRD-TMH-001, was meant use the CRISPR gene editing tool to increase the expression of an alternate form of the dystrophin protein. These would ameliorate the issues caused by mutations in the promotor region and exon 1 of the dystrophin gene. CRISPR can be used to instruct a cell to skip certain exons when a protein is being made, such as the exons that contain mutations.” Unfortunately, the sole patient in this trial died. MORE
Image Credit. STAT