Gapmer Antisense Oligonucleotides Suppress the Mutant Allele of COL6A3 and Restore Functional Protein in Ullrich Muscular Dystrophy
Gapmer Antisense Oligonucleotides Suppress the Mutant Allele of COL6A3 and Restore Functional Protein in Ullrich Muscular Dystrophy …………… Marrosu E, Ala P, Muntoni F, Zhou H. Mol Ther Nucleic Acids. 2017 Sep 15;8:416-427. PubMed 00