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: Claire Domenger, Marine Allais, Virginie François, Adrien Léger, Emilie Lecomte, Marie Montus, Laurent Servais, Thomas Voit, Philippe Moullier, Yann Audic, Caroline Le Guiner
Format: Article
Language:English
Published: Elsevier 2018-03-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
exon skipping
off-target
RNA sequencing
Duchenne
U7snRNA
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253117303074

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http://www.sciencedirect.com/science/article/pii/S2162253117303074

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