New advancements in CRISPR based gene therapy of Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is caused by the dystrophin gene mutations and is one of the most common and lethal human hereditary disorders. A novel therapeutic approach using CRISPR technology has gained attention in the treatment of DMD. Gene replacement strategies are being proposed as a promising therapeutic option to compensate the loss of function mutations. Although, the large size of the dystrophin gene and the limitations of the existing gene replacement approach, could mean the gene delivery of shortened versions of dystrophin such as midystrophin and microdystrophins. There are also other approaches: including Targeted removal of dystrophin exons to restore the reading-frame; Dual sgRNA-directed DMD exon deletion, CRISPR-SKIP strategy; reframing of dystrophin using Prime Editing technology; exon removal using twin prime tech-nology; TransCRISTI technology to targeted exon integration into dystrophin gene. Here we provide an overview of recent progresses in dystrophin gene editing using updated versions of CRISPR to introduce novel opportu-nities in DMD gene therapy.Overall, the novel CRISPR based technologies are improving and expanding to allow the application of more precise gene editing for the treatment of DMD.

Automated summary

Duchenne muscular dystrophy is a common and lethal genetic disorder caused by mutations in the dystrophin gene. CRISPR technology has emerged as a promising therapeutic approach for treating this disease. Gene replacement strategies, such as using shortened versions of dystrophin, have been proposed to compensate for loss-of-function mutations.