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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Detalles Bibliográficos
Autores principales: Gallego-Villar, Lorena, Viecelli, Hiu Man, Pérez, Belén, Harding, Cary O, Ugarte, Magdalena, Thöny, Beat, Desviat, Lourdes R
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2014
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4222650/
https://www.ncbi.nlm.nih.gov/pubmed/25226162
http://dx.doi.org/10.1038/mtna.2014.44

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4222650/
https://www.ncbi.nlm.nih.gov/pubmed/25226162
http://dx.doi.org/10.1038/mtna.2014.44

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