Myotonic muscular dystrophy, RNA toxicity, and the brain: trouble making the connection?
basic_science · Level V
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- Record sourced from PubMed, PMID 21474094.
- Also identified by DOI 10.1016/j.stem.2011.03.007.
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Abstract
The study of rare genetic diseases is complicated by the inaccessibility of relevant cells and tissues, especially for neurologic disorders. In this issue of Cell Stem Cell, Marteyn et al. (2011) use human embryonic stem cells to identify deficits in neuritic outgrowth in myotonic dystrophy type 1.