Gapmer Antisense Oligonucleotides to Selectively Suppress the Mutant Allele in COL6A Genes in Dominant Ullrich Congenital Muscular Dystrophy.
Sign Up to like & getrecommendations! Published in 2020 at "Methods in molecular biology"
DOI: 10.1007/978-1-0716-0771-8_16
Abstract: Allele-specific gene silencing by antisense oligonucleotide (ASO) or small interference RNA (siRNA) has been used as a therapeutic approach for conditions caused by dominant gain-of-function mutations. We here present an antisense approach using gapmer ASO… read more here.
Keywords: ullrich congenital; congenital muscular; muscular dystrophy; col6a genes ... See more keywords