Article
Repeat instability as the basis for human diseases and as a potential target for therapy.
Nature reviews. Molecular cell biology - 1 Mar 2010
López Castel Arturo, Cleary John D, Pearson Christopher E
Abstract excerpt
Expansions of repetitive DNA sequences cause numerous human neurological and neuromuscular diseases. Ongoing repeat expansions in patients can exacerbate disease progression and severity. As pathogenesis is connected to repeat length, a potential therapeutic avenue is to modulate disease by manipulating repeat expansion size--targeting DNA, the root-cause of symptoms. How repeat instability is mediated by DNA...
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