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Article

AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models

20 Jan 2015

Abstract excerpt

OBJECTIVE: Dysferlinopathies are a family of untreatable muscle disorders caused by mutations in the dysferlin gene. Lack of dysferlin protein results in progressive dystrophy with chronic muscle fiber loss, inflammation, fat replacement, and fibrosis; leading to deteriorating muscle weakness. The objective of this work is to demonstrate efficient and safe restoration of dysferlin expression following gene...

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PMID: 25815352
DOI: 10.1002/acn3.172

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