RNA-Seq Analysis of an Antisense Sequence Optimized for Exon Skipping in Duchenne Patients Reveals No Off-Target Effect

• Main Authors: Claire Domenger (Author), Marine Allais (Author), Virginie François (Author), Adrien Léger (Author), Emilie Lecomte (Author), Marie Montus (Author), Laurent Servais (Author), Thomas Voit (Author), Philippe Moullier (Author), Yann Audic (Author), Caroline Le Guiner (Author)
• Format: Bog
• Udgivet: Elsevier, 2018-03-01T00:00:00Z.
• Fag: article
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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)...