In Vitro Correction of a Pseudoexon-Generating Deep Intronic Mutation in LGMD2A by Antisense Oligonucleotides and Modified Small Nuclear RNAs
Author:
Affiliation:
1. Center for Genomic Regulation (CRG); Barcelona; Spain
2. Université Pierre et Marie Curie; Paris; France
3. Center for Applied Medical Research (CIMA); University of Navarra; Pamplona; Spain
Publisher
Wiley
Subject
Genetics (clinical),Genetics
Link
http://onlinelibrary.wiley.com/wol1/doi/10.1002/humu.22379/fullpdf
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3. Characterization of novel CAPN3 isoforms in white blood cells: an alternative approach for limb-girdle muscular dystrophy 2A diagnosis;Blazquez;Neurogenetics,2008
4. Increased in vivo inhibition of gene expression by combining RNA interference and U1 inhibition;Blazquez;Nucleic Acids Res,2012
5. U7 snRNAs induce correction of mutated dystrophin pre-mRNA by exon skipping;Brun;Cell Mol Life Sci,2003
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