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

Collagen VI-related congenital muscular dystrophies (COL6-CMDs) are the second most common form of congenital muscular dystrophy. Currently, there is no effective treatment available. COL6-CMDs are caused by recessive or dominant mutations in one of the three genes encoding for the α chains of colla...

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Veröffentlicht in:Mol Ther Nucleic Acids
Hauptverfasser: Aguti, Sara, Bolduc, Véronique, Ala, Pierpaolo, Turmaine, Mark, Bönnemann, Carsten G., Muntoni, Francesco, Zhou, Haiyan
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Society of Gene & Cell Therapy 2020
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Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7321786/
https://ncbi.nlm.nih.gov/pubmed/32585628
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2020.05.029
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