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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書誌詳細

出版年:	Molecular Therapy: Nucleic Acids
主要な著者:	Lorena Gallego-Villar, Hiu Man Viecelli, Belén Pérez, Cary O Harding, Magdalena Ugarte, Beat Thöny, Lourdes R Desviat
フォーマット:	論文
言語:	英語
出版事項:	Elsevier 2014-01-01
主題:	animal models antisense oligonucleotides exon skipping hyperphenylalaninemia metabolic diseases splicing suppression vivo-morpholino
オンライン･アクセス:	http://www.sciencedirect.com/science/article/pii/S2162253116303328