Exon-Skipping Oligonucleotides Restore Functional Collagen VI by Correcting a Common COL6A1 Mutation in Ullrich CMD

Published 2020 View Full Article

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Title
Exon-Skipping Oligonucleotides Restore Functional Collagen VI by Correcting a Common COL6A1 Mutation in Ullrich CMD
Authors
Keywords
Collagen VI, congenital muscular dystrophy, Ullrich muscular dystrophy, deep intronic mutation, exon skipping, antisense oligonucleotide, extra cellular matrix
Journal
Molecular Therapy-Nucleic Acids
Volume 21, Issue -, Pages 205-216
Publisher
Elsevier BV
Online
2020-06-02
DOI
10.1016/j.omtn.2020.05.029

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