RNA-seq analysis of an antisense sequence optimized for exon skipping in Duchenne patients reveals no off-target effect

RNA-seq analysis of an antisense sequence optimized for exon skipping in Duchenne patients reveals no off-target effect

Non-coding uridine-rich small nuclear RNAs (UsnRNAs) have emerged in recent years as effective tools for exon skipping for the treatment of Duchenne muscular dystrophy (DMD), a degenerative muscular genetic disorder. We recently showed the high capacity of a recombinant adeno-associated virus (rAAV)...

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Bibliographic Details
Main Authors:	Domenger, C, Allais, M, François, V, Léger, A, Lecomte, E, Montus, M, Servais, L, Voit, T, Moullier, P, Audic, Y, Le Guiner, C
Format:	Journal article
Language:	English
Published:	Elsevier 2017

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