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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Detalles Bibliográficos
Autores principales: Domenger, Claire, Allais, Marine, François, Virginie, Léger, Adrien, Lecomte, Emilie, Montus, Marie, Servais, Laurent, Voit, Thomas, Moullier, Philippe, Audic, Yann, Le Guiner, Caroline
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2017
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5785776/
https://www.ncbi.nlm.nih.gov/pubmed/29499940
http://dx.doi.org/10.1016/j.omtn.2017.12.008

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