Published in Gene Therapy Weekly, November 23rd, 2006
Study 1: A study from the United States has reported on a reversible model of RNA toxicity and cardiac conduction defects in myotonic dystrophy (DM1).
"DM1, the most common muscular dystrophy in adults, is caused by an expanded (CTG)(n) tract in the 3' UTR of the gene encoding myotonic dystrophy protein kinase (DMPK) 1, which results in nuclear entrapment of the 'toxic' mutant RNA and interacting RNA-binding proteins (such as MBNL1) in ribonuclear inclusions.
"It is unclear if therapy aimed at eliminating the toxin would be beneficial. To address...
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Source: Gene Therapy Weekly (2006-11-23)
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