The paper titled "Multiple Exon Skipping in the Duchenne Muscular Dystrophy Hot Spots: Prospects and Challenges" explores an advanced therapeutic strategy for treating Duchenne Muscular Dystrophy (DMD), a severe genetic disorder caused by mutations in the dystrophin gene. This mutation leads to the absence of functional dystrophin protein, which is essential for maintaining muscle integrity. One emerging treatment method involves antisense oligonucleotide (AO)-mediated exon skipping, where specific exons are skipped …

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Personal take on this article:   Summary: Duchenne muscular dystrophy (DMD) is a severe, X-linked disorder characterized by progressive muscle wasting due to mutations in the dystrophin gene. Exon-skipping therapy, utilizing antisense oligonucleotides (AOs), aims to bypass mutated exons, restoring the reading frame and enabling the production of functional dystrophin, thereby converting DMD to a milder Becker muscular dystrophy (BMD) phenotype.   1. Introduction:   - DMD is caused by …

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