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Article

Quantitative Antisense Screening and Optimization for Exon 51 Skipping in Duchenne Muscular Dystrophy.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Nov 2017

Echigoya Yusuke, Lim Kenji Rowel Q, Trieu Nhu, Bao Bo, Miskew Nichols Bailey, Vila Maria Candida, Novak James S, Hara Yuko, Lee Joshua, Touznik Aleksander, Mamchaoui Kamel, Aoki Yoshitsugu, Takeda Shin'ichi, Nagaraju Kanneboyina, Mouly Vincent, Maruyama Rika, Duddy William, Yokota Toshifumi

Abstract excerpt

Duchenne muscular dystrophy (DMD), the most common lethal genetic disorder, is caused by mutations in the dystrophin (DMD) gene. Exon skipping is a therapeutic approach that uses antisense oligonucleotides (AOs) to modulate splicing and restore the reading frame, leading to truncated, yet functional protein expression. In 2016, the US Food and Drug Administration (FDA) conditionally approved the first...

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Source

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Identifiers

PMID: 28865998
DOI: 10.1016/j.ymthe.2017.07.014
PMCID: PMC5675502

Genes

DMD

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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