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Article

Full-length dysferlin expression driven by engineered human dystrophic blood derived CD133+ stem cells.

The FEBS journal - 1 Dec 2013

Meregalli Mirella, Navarro Claire, Sitzia Clementina, Farini Andrea, Montani Erica, Wein Nicolas, Razini Paola, Beley Cyriaque, Cassinelli Letizia, Parolini Daniele, Belicchi Marzia, Parazzoli Dario, Garcia Luis, Torrente Yvan

Abstract excerpt

The protein dysferlin is abundantly expressed in skeletal and cardiac muscles, where its main function is membrane repair. Mutations in the dysferlin gene are involved in two autosomal recessive muscular dystrophies: Miyoshi myopathy and limb-girdle muscular dystrophy type 2B. Development of effective therapies remains a great challenge. Strategies to repair the dysferlin gene by skipping mutated exons, using...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 24028392
DOI: 10.1111/febs.12523

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 5 Sept 2026. Manifest publication-graph-cohort-2026-09-05.1.

Source receipt: pubmed, retrieved 5 Sept 2026.

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