CRISPR/Cas9-mediated gene correction of Wilson disease H1069Q point mutation in patient-specific induced pluripotent stem cells
Vanessa Sandfort
Gene Therapy, Published online: 14 April 2026; doi:10.1038/s41434-026-00611-7 CRISPR/Cas9-mediated gene correction of Wilson disease H1069Q point mutation in patient-specific induced pluripotent stem cells