Article
A TALEN-Exon Skipping Design for a Bethlem Myopathy Model in Zebrafish.
PloS one - 1 Jan 2015
Radev Zlatko, Hermel Jean-Michel, Elipot Yannick, Bretaud Sandrine, Arnould Sylvain, Duchateau Philippe, Ruggiero Florence, Joly Jean-Stéphane, Sohm Frédéric
Abstract excerpt
Presently, human collagen VI-related diseases such as Ullrich congenital muscular dystrophy (UCMD) and Bethlem myopathy (BM) remain incurable, emphasizing the need to unravel their etiology and improve their treatments. In UCMD, symptom onset occurs early, and both diseases aggravate with ageing. In zebrafish fry, morpholinos reproduced early UCMD and BM symptoms but did not allow to study the late phenotype....
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