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Myotonic dystrophy type 1 and splice-blocking oligos

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jonmoulton

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Morpholinos targeting exon 7a of the muscle-specific chloride channel 1 (ClC-1) relieved symptoms of myotonic dystrophy type 1 in two mouse models.

Wheeler TM, Lueck JD, Swanson MS, Dirkson RT, Thornton CA. Correction of CIC-1 splicing eliminates chloride channelopathy and myotonia in mouse models of myotonic dystrophy. JCI 2007 doi:10.1172/JCI33355

https://www.the-jci.org/article.php?id=33355
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Jon D. Moulton, Ph.D. Gene Tools, LLC www.gene-tools.com
Posted Nov 16, 2007, 20:45 PM
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