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...

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Detalles Bibliográficos
Autores principales: Marrosu, Elena, Ala, Pierpaolo, Muntoni, Francesco, Zhou, Haiyan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2017
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5537204/
https://www.ncbi.nlm.nih.gov/pubmed/28918041
http://dx.doi.org/10.1016/j.omtn.2017.07.006

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5537204/
https://www.ncbi.nlm.nih.gov/pubmed/28918041
http://dx.doi.org/10.1016/j.omtn.2017.07.006

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