Which therapeutic category includes eteplirsen, golodirsen, viltolarsen, and casimersen?

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Prepare for the Muscular Dystrophy Test with comprehensive flashcards and multiple-choice questions. Access detailed hints and explanations for each question to enhance your study experience. Equip yourself for success on your upcoming exam!

Multiple Choice

Which therapeutic category includes eteplirsen, golodirsen, viltolarsen, and casimersen?

Explanation:
Exon-skipping therapies use antisense oligonucleotides to bind dystrophin pre-mRNA and hide a specific exon from the splicing machinery. By skipping that exon during mRNA processing, the downstream reading frame can be restored, producing a shorter but still functional dystrophin protein rather than a completely disrupted one. This approach is tailored to Duchenne muscular dystrophy mutations where removing a particular exon reestablishes the open reading frame. Eteplirsen targets exon 51, golodirsen and viltolarsen target exon 53, and casimersen targets exon 45. This makes them all exon-skipping therapies, rather than gene-silencing, immunotherapies, or stem cell therapies.

Exon-skipping therapies use antisense oligonucleotides to bind dystrophin pre-mRNA and hide a specific exon from the splicing machinery. By skipping that exon during mRNA processing, the downstream reading frame can be restored, producing a shorter but still functional dystrophin protein rather than a completely disrupted one. This approach is tailored to Duchenne muscular dystrophy mutations where removing a particular exon reestablishes the open reading frame.

Eteplirsen targets exon 51, golodirsen and viltolarsen target exon 53, and casimersen targets exon 45. This makes them all exon-skipping therapies, rather than gene-silencing, immunotherapies, or stem cell therapies.

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