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In Vitro Correction of a Pseudoexon‐Generating Deep Intronic Mutation in LGMD2A by Antisense Oligonucleotides and Modified Small Nuclear RNAs |
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| Authors: | Lorea Blázquez Ana Aiastui Maria Goicoechea Mafalda Martins de Araujo Aurélie Avril Cyriaque Beley Luis García Juan Valcárcel Adolfo López de Munain |
| Affiliation: | 1. Center for Applied Medical Research (CIMA), University of Navarra, , Pamplona, Spain;2. Neuroscience Area, Health Research Institute Biodonostia, , San Sebastian, Spain;3. CIBERNED, Carlos III Institute, Ministry of Economy and Competitiveness, , Madrid, Spain;4. Center for Genomic Regulation (CRG), , Barcelona, Spain;5. Université Pierre et Marie Curie, , Paris, France |
| Abstract: | |
| Keywords: | limb‐girdle muscular dystrophy LGMD2A pseudoexon exon skipping antisense oligonucleotide AON usnRNA |