Mice Lacking the Myotonic Dystrophy Protein Kinase Develop a Late Onset Progressive Myopathy

Document Type

Article

Publication Date

7-1-1996

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Abstract

Myotonic dystrophy (DM) is an autosomal dominant disorder resulting from the expansion of a CTG repeat in the 3′ untranslated region of a putative protein kinase (DMPK). To elucidate the role of DMPK in DM pathogenesis we have developed DMPK deficient (DMPK−/−) mice. DMPK−/− mice develop a late-onset, progressive skeletal myopathy that shares some pathological features with DM. Muscles from mature mice show variation in fibre size, increased fibre degeneration and fibrosis. Adult DMPK−/− mice show ultrastructural changes in muscle and a 50% decrease in force generation compared to young mice. Our results indicate that DMPK may be necessary for the maintenance of skeletal muscle structure and function and suggest that a decrease in DMPK levels may contribute to DM pathology.

DOI

10.1038/ng0796-325

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