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Article

Efficient exon skipping of SGCG mutations mediated by phosphorodiamidate morpholino oligomers.

JCI insight - 3 May 2018

Wyatt Eugene J, Demonbreun Alexis R, Kim Ellis Y, Puckelwartz Megan J, Vo Andy H, Dellefave-Castillo Lisa M, Gao Quan Q, Vainzof Mariz, Pavanello Rita C M, Zatz Mayana, McNally Elizabeth M

Abstract excerpt

Exon skipping uses chemically modified antisense oligonucleotides to modulate RNA splicing. Therapeutically, exon skipping can bypass mutations and restore reading frame disruption by generating internally truncated, functional proteins to rescue the loss of native gene expression. Limb-girdle muscular dystrophy type 2C is caused by autosomal recessive mutations in the SGCG gene, which encodes the...

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Identifiers

PMID: 29720576
DOI: 10.1172/jci.insight.99357
PMCID: PMC6012523

Genes

SGCG

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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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