Genome editing is an emerging approach to the targeted treatment of Duchenne muscular dystrophy (DMD). Japanese researchers have conducted experiments using the CRISPR-Cas9 tool in induced pluripotent stem cells (iPSc):
- unlike other genome-editing tools that target one exon of the DMD gene, the system used can skip many exons at once, up to a total portion of 340 kilobases of DNA,
- no genomic perturbations were observed outside the targeted areas (off-targeting)
- this approach, known as MES for Multiple Exon Skipping, makes it possible to restore the reading frame of the DMD gene and produce a partially functional dystrophin,
- its application in humans is currently being studied.
