Dyne is eyeing an accelerated approval filing for DYNE-251 in early 2026 that would pit the asset against Sarepta’s Exondys ...

The large DMD gene has 79 exons. For DMD patients with suitable mutations, exon skipping is one way to convert out-of-frame mRNA translation to a properly aligned reading frame. Oligonucleotides ...

The DMD gene is made up of 79 exons, and mutations in that code can result in a deficiency in dystrophin which is responsible for the muscle wasting in DMD. Exon-skipping drugs are used to patch ...

The DMD gene is made up of 79 exons, and mutations in that code can result in a deficiency in dystrophin which is responsible for the muscle wasting in DMD. Exon-skipping drugs are used to patch ...

DMD mutations are particularly common in “hotspot” areas of the gene (exons 45–55 and 2–10). Because the genetic underpinnings of Duchenne are known, researchers can devise gene-editing fixes to the ...

Exons are coding sections of an RNA transcript, or the DNA encoding it, that are translated into protein. Exons can be separated by intervening sections of DNA that do not code for proteins, known ...

Three of its drugs skip exons — or specific places in the genome ... "First, exon 44 skipping represents less than 5% of the ...

Presentation Details: Title: ARCUS-Mediated Excision of Exons 45-55 Leads to Functional Del45-55 Dystrophin and Restoration of Skeletal Muscle-Function for the Treatment of DMD Date and Time ...

Duchenne muscular dystrophy (DMD) is a fatal genetic disorder caused ... Non-viral intron knock-ins for incorporating synthetic exons into endogenous introns enable efficient gene targeting ...