A Sensitive Assay System To Test Antisense Oligonucleotides for Splice Suppression Therapy in the Mouse Liver

We have previously demonstrated the efficacy of antisense therapy for splicing defects in cellular models of metabolic diseases, suppressing the use of cryptic splice sites or pseudoexon insertions. To date, no animal models with these defects are available. Here, we propose exon skipping of the phe...

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מידע ביבליוגרפי

Principais autores:	Lorena Gallego-Villar, Hiu Man Viecelli, Belén Pérez, Cary O Harding, Magdalena Ugarte, Beat Thöny, Lourdes R Desviat
פורמט:	Artigo
שפה:	Inglês
יצא לאור:	Elsevier 2014-01-01
סדרה:	Molecular Therapy: Nucleic Acids
נושאים:	animal models antisense oligonucleotides exon skipping hyperphenylalaninemia metabolic diseases splicing suppression vivo-morpholino
גישה מקוונת:	http://www.sciencedirect.com/science/article/pii/S2162253116303328
תגים:	הוספת תג אין תגיות, היה/י הראשונ/ה לתייג את הרשומה!