Telomere position effect regulates DUX4 in human facioscapulohumeral muscular dystrophy
Stadler, Guido ; Rahimov, Fedik ; King, Oliver D. ; Chen, Jennifer C. J. ; Robin, Jerome D. ; Wagner, Kathryn R. ; Shay, Jerry W. ; Emerson, Charles P. Jr. ; Wright, Woodring E.
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Abstract
Telomeres may regulate human disease by at least two independent mechanisms. First, replicative senescence occurs once short telomeres generate DNA-damage signals that produce a barrier to tumor progression. Second, telomere position effects (TPE) could change gene expression at intermediate telomere lengths in cultured human cells. Here we report that telomere length may contribute to the pathogenesis of facioscapulohumeral muscular dystrophy (FSHD). FSHD is a late-onset disease genetically residing only 25-60 kilobases from the end of chromosome 4q. We used a floxable telomerase to generate isogenic clones with different telomere lengths from affected patients and their unaffected siblings. DUX4, the primary candidate for FSHD pathogenesis, is upregulated over ten-fold in FSHD myoblasts and myotubes with short telomeres, and its expression is inversely proportional to telomere length. FSHD may be the first known human disease in which TPE contributes to age-related phenotype.
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Stadler G, Rahimov F, King OD, Chen JC, Robin JD, Wagner KR, Shay JW, Emerson CP Jr, Wright WE. Telomere position effect regulates DUX4 in human facioscapulohumeral muscular dystrophy. Nat Struct Mol Biol. 2013 Jun;20(6):671-8. doi: 10.1038/nsmb.2571. Link to article on publisher's site