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Article

Adenoviral vectors encoding CRISPR/Cas9 multiplexes rescue dystrophin synthesis in unselected populations of DMD muscle cells.

Scientific reports - 15 Nov 2016

Maggio Ignazio, Liu Jin, Janssen Josephine M, Chen Xiaoyu, Gonçalves Manuel A F V

Abstract excerpt

Mutations disrupting the reading frame of the ~2.4 Mb dystrophin-encoding DMD gene cause a fatal X-linked muscle-wasting disorder called Duchenne muscular dystrophy (DMD). Genome editing based on paired RNA-guided nucleases (RGNs) from CRISPR/Cas9 systems has been proposed for permanently repairing faulty DMD loci. However, such multiplexing strategies require the development and testing of delivery systems...

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Share to Adenoviridae
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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Dystrophin

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Share to Muscular Dystrophy, Duchenne

Source

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Identifiers

PMID: 27845387
DOI: 10.1038/srep37051
PMCID: PMC5109245

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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