Exon-Skipping Oligonucleotides Restore Functional Collagen VI by Correcting a Common COL6A1 Mutation in Ullrich CMD.
Molecular Therapy — Nucleic Acids（2020）
关键词
Collagen VI,congenital muscular dystrophy,Ullrich muscular dystrophy,deep intronic mutation,exon skipping,antisense oligonucleotide,extra cellular matrix
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