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Article

Reengineering a transmembrane protein to treat muscular dystrophy using exon skipping.

The Journal of clinical investigation - 2 Nov 2015

Gao Quan Q, Wyatt Eugene, Goldstein Jeff A, LoPresti Peter, Castillo Lisa M, Gazda Alec, Petrossian Natalie, Earley Judy U, Hadhazy Michele, Barefield David Y, Demonbreun Alexis R, Bönnemann Carsten, Wolf Matthew, McNally Elizabeth M

Abstract excerpt

Exon skipping uses antisense oligonucleotides as a treatment for genetic diseases. The antisense oligonucleotides used for exon skipping are designed to bypass premature stop codons in the target RNA and restore reading frame disruption. Exon skipping is currently being tested in humans with dystrophin gene mutations who have Duchenne muscular dystrophy. For Duchenne muscular dystrophy, the rationale for exon...

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Source

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Identifiers

PMID: 26457733
DOI: 10.1172/JCI82768
PMCID: PMC4639981

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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