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

por Aguti, Sara, Bolduc, Véronique, Ala, Pierpaolo, Turmaine, Mark, Bönnemann, Carsten G., Muntoni, Francesco, Zhou, Haiyan
Publicado 2020