Exon skipping therapy generally aims to convert out-of-frame mutations in DMD patients to in-frame variants. A single CRISPR/Cas9 system can fix the exon 2 duplication in DMD myoblasts.
In a different study, defective exon 23 was removed using the CRISPR-Cas9 system in dystrophic animals. The results showed that genome editing restored the expression of dystrophin. Gene transfer to restore dystrophin expression utilising the secure, non-pathogenic adeno-associated viral (AAV) vector is a potential method for curing this fatal condition. CRISPR gene editing techniques for treating DMD can be developed to reduce the loss of genomic DNA. One can create a single sgRNA to abolish either the splice or the mutation rather than employing two sgRNAs flanking a mutant exon.
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