Myotonic muscular dystrophy, RNA toxicity, and the brain: trouble making the connection?

Mahadevan, Mani S · Cell Stem Cell · 2011

basic_science · Level V

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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.