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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   This article is simplified from English Wikipedia. https://en.wikipedia.org/wiki/Exon_skipping  Exon skipping is a molecular biology technique used to address genetic mutations by causing cells to bypass defective sections (exons) of the genetic code. This process involves manipulating RNA splicing, specifically targeting mutations in the pre-messenger RNA.    Genes contain coding regions called exons, which provide instructions for protein creation, separated by non-coding introns. Exon skipping aims to restore the reading …

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