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Article

Selection-free gene repair after adenoviral vector transduction of designer nucleases: rescue of dystrophin synthesis in DMD muscle cell populations.

Nucleic acids research - 18 Feb 2016

Maggio Ignazio, Stefanucci Luca, Janssen Josephine M, Liu Jin, Chen Xiaoyu, Mouly Vincent, Gonçalves Manuel A F V

Abstract excerpt

Duchenne muscular dystrophy (DMD) is a fatal X-linked muscle-wasting disorder caused by mutations in the 2.4 Mb dystrophin-encoding DMD gene. The integration of gene delivery and gene editing technologies based on viral vectors and sequence-specific designer nucleases, respectively, constitutes a potential therapeutic modality for permanently repairing defective DMD alleles in patient-derived myogenic cells....

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Topics

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Share to Adenoviridae
Share to DNA End-Joining Repair

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Dystrophin

Share to Endonucleases

Share to Muscular Dystrophy, Duchenne

Share to Myoblasts

Share to RNA, Guide, CRISPR-Cas Systems

Share to Transduction, Genetic

Source

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Identifiers

PMID: 26762977
DOI: 10.1093/nar/gkv1540
PMCID: PMC4756843

Genes

DMD
RGN

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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