Effective Exon Skipping and Restoration of Dystrophin Expression by Peptide Nucleic Acid Antisense Oligonucleotides in mdx Mice
Author:
Publisher
Elsevier BV
Subject
Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine
Reference28 articles.
1. Subcellular fractionation of dystrophin to the triads of skeletal muscle;Hoffman;Nature,1987
2. Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides;Dunckley;Hum Mol Genet,1998
3. Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells;Van Deutekom;Hum Mol Genet,2001
4. Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides;Wilton;Neuromuscul Disord,1999
5. Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse;Mann;Proc Natl Acad Sci USA,2001
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