Gapmer Antisense Oligonucleotides Suppress the Mutant Allele of COL6A3 and Restore Functional Protein in Ullrich Muscular Dystrophy

Dominant-negative mutations in the genes that encode the three major α chains of collagen type VI, COL6A1, COL6A2, and COL6A3, account for more than 50% of Ullrich congenital muscular dystrophy patients and nearly all Bethlem myopathy patients. Gapmer antisense oligonucleotides (AONs) are usually us...

पूर्ण विवरण

में बचाया:
ग्रंथसूची विवरण
में प्रकाशित:Mol Ther Nucleic Acids
मुख्य लेखकों: Marrosu, Elena, Ala, Pierpaolo, Muntoni, Francesco, Zhou, Haiyan
स्वरूप: Artigo
भाषा:Inglês
प्रकाशित: American Society of Gene & Cell Therapy 2017
विषय:
Original Article
ऑनलाइन पहुंच:https://ncbi.nlm.nih.gov/pmc/articles/PMC5537204/
https://ncbi.nlm.nih.gov/pubmed/28918041
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2017.07.006
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