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SaveMyosin inhibition partially rescues the myofiber proteome in X-linked myotubular myopathy
Melhedegaard, Elise Gerlach; Rostedt, Fanny; Gineste, Charlotte; Seaborne, Robert A. E.; Dugdale, Hannah F.; Belhac, Vladimir; Zanoteli, Edmar; Lawlor, Michael W.; Mack, David L.; Wallgren-Pettersson, Carina; Hessel, Anthony L.; Jungbluth, Heinz; Laporte, Jocelyn; Saito, Yoshihiko; Nishino, Ichizo; Ochala, Julien; Laitila, Jenni
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SaveSubstitutions of nucleotides at the 3′ ends of COL6A1/2/3 exons induce exon skipping associated with collagen VI-related muscular dystrophies and therapeutic strategies
Lee, Seung-Ah; Ogawa, Megumu; Saito, Yoshihiko; Shimazaki, Rui; Awaya, Tomonari; Hosokawa, Motoyasu; Kurosawa, Ryo; Ohara, Hiroaki; Takeuchi, Akihide; Hayashi, Shinichiro; Goto, Yu-ichi; Hagiwara, Masatoshi; Nishino, Ichizo; Noguchi, Satoru
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