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

• Main Authors: Lorena Gallego-Villar (Author), Hiu Man Viecelli (Author), Belén Pérez (Author), Cary O Harding (Author), Magdalena Ugarte (Author), Beat Thöny (Author), Lourdes R Desviat (Author)
• Format: Book
• Published: Elsevier, 2014-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...