Duchenne muscular dystrophy is an X-linked recessive hereditary monogenic disorder caused by inability to produce dystrophin protein. In most patients

the expression of dystrophin lost due to disrupting mutations in open reading frame. Despite the efforts in a large number of different therapeutic approaches to date

the treatments available for DMD remain mitigative and supportive to improve the symptoms of the disease

rather than to be curative. The advent of CRISPR/Cas9 technology has revolutionized genome editing scope and considered as pioneer in effective genomic engineering. Deletions or excisions of intragenic DNA by CRISPR as well as a similar strategy with exon skipping at the DNA level induced by antisense oligonucleotides

are new and promising approaches in correcting

DMD

gene

which restore the expression of a truncated but functional dystrophin protein. Also

CRISPR/Cas9 technology can be used to treat DMD by removing duplicated exons

precise correction of causative mutation by HDR-based pathway and inducing the expression of compensatory proteins such as utrophin. In this study

we briefly explained the molecular genetics of DMD and a historical overview of DMD gene therapy. We in particular focused on CRISPR/Cas9-mediated therapeutic approaches that used to treat DMD.