Title: | Gapmer Antisense Oligonucleotides Suppress the Mutant Allele of COL6A3 and Restore Functional Protein in Ullrich Muscular Dystrophy |
Journal : | Molecular therapy. Nucleic acids, 8 |
Authors: | Marrosu E, Author ; Ala P ; Muntoni F ; Zhou H |
Material Type: | Article |
Publication Date: | 2017 |
Size: | p 416 |
Languages: | English |
Keywords : | allele silencing ; antisense oligonucleotides ; cell lines ; COL6 gene ; collagen VI ; collagen VI related disease ; extracellular matrix ; fibroblasts ; gapmer ; gene expression ; genetic mutations ; messenger RNA ; Ullrich congenital muscular dystrophy |
Keywords: | allele-specific silencing;antisense oligonucleotide therapy;collagen VI;congenital muscular dystrophy;dominant mutations;gapmer; |
Pubmed / DOI : | DOI : 10.1016/j.omtn.2017.07.006 / Pubmed : 28918041 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/28918041 |
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