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Article

An Updated Analysis of Exon-Skipping Applicability for Duchenne Muscular Dystrophy Using the UMD-DMD Database.

Genes - 20 Nov 2024

Leckie Jamie, Zia Abdullah, Yokota Toshifumi

Abstract excerpt

BACKGROUND/OBJECTIVES: Antisense oligonucleotide (ASO)-mediated exon-skipping is an effective approach to restore the disrupted reading frame of the dystrophin gene for the treatment of Duchenne muscular dystrophy (DMD). Currently, four FDA-approved ASOs can target three different exons, but these therapies are mutation-specific and only benefit a subset of patients. Understanding the broad applicability of...

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Share to Databases, Genetic
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Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

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Share to Muscular Dystrophy, Duchenne

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Share to Oligonucleotides, Antisense

Source

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Identifiers

PMID: 39596689
DOI: 10.3390/genes15111489
PMCID: PMC11593839

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