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

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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Bibliographic Details
Main Authors: Lorena Gallego-Villar, Hiu Man Viecelli, Belén Pérez, Cary O Harding, Magdalena Ugarte, Beat Thöny, Lourdes R Desviat
Format: Article
Language:English
Published: Elsevier 2014-01-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
animal models
antisense oligonucleotides
exon skipping
hyperphenylalaninemia
metabolic diseases
splicing suppression
vivo-morpholino
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253116303328

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http://www.sciencedirect.com/science/article/pii/S2162253116303328

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