A CRISPR-Cas9 system induces multiple exon skipping in DMD

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 exonPartie codante de l’ADN au sein d’un gène. of the DMD gene, the system used can skip many exons at once, up to a total portion of 340 kilobases of DNAmacromolécule complexe, l’ADN est le support de l’hérédité (gènes). C’est le constituant des chromosomes. L’ADN est organisé en double hélice (deux brins complémentaires) et constitué de nucléotides de quatre types : adénine, guanine, cytosine et thymine.,
• 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.

Dual CRISPR-Cas3 system for inducing multi-exon skipping in DMD patient-derived iPSCs. Kita Y, Okuzaki Y, Naoe Y et al. Stem Cell Reports. 2023 Sep