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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...

Topics

Humans
Databases, Genetic
Dystrophin
Exons
Genetic Therapy
Muscular Dystrophy, Duchenne
Mutation
Oligonucleotides, Antisense

Source

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Identifiers

PMID: 39596689
DOI: 10.3390/genes15111489
PMCID: PMC11593839

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 31 Aug 2026.

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