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Article

Dysferlin overexpression in skeletal muscle produces a progressive myopathy.

Annals of neurology - 1 Mar 2010

Glover Louise E, Newton Kimberly, Krishnan Gomathi, Bronson Roderick, Boyle Alexandra, Krivickas Lisa S, Brown Robert H

Abstract excerpt

OBJECTIVE: The dose-response effects of dysferlin transgenesis were analyzed to determine if the dysferlin-deficient myopathies are good candidates for gene replacement therapy. METHODS: We have generated 3 lines of transgenic mice, expressing low, mid, and high levels of full-length human dysferlin from a muscle-specific promoter. Transgenic skeletal muscle was analyzed and scored for morphological and...

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Identifiers

PMID: 20373350
DOI: 10.1002/ana.21926
PMCID: PMC3900233

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