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. 2016 Jan 26;5(1):e283. doi: 10.1038/mtna.2015.58

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Copyright © 2016 Official journal of the American Society of Gene & Cell Therapy

This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivs 4.0 International License. The images or other third party material in this article are included in the article's Creative Commons license, unless indicated otherwise in the credit line; if the material is not included under the Creative Commons license, users will need to obtain permission from the license holder to reproduce the material. To view a copy of this license, visit http://creativecommons.org/licenses/by-nc-nd/4.0/

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CinDel correction in myoblasts restored the DYS protein expression in myotubes. (a) Normal wild-type myoblasts (CTL+), uncorrected DMD myoblasts with a deletion of exons 51–53 (CTL-) as well as CinDel-corrected DMD myoblasts (CinDel) were allowed to fuse to form abundant myotubes containing multiple nuclei. Proteins were extracted from these three types of myotubes. The DMD myoblasts (Δ51–53) were genetically corrected with (b) gRNA2–50 and gRNA2–54 and (c) with gRNA1–50 and gRNA5–54. In b and c, western blot detected no DYS protein in uncorrected DMD myotubes (CTL-), a 427 kDa DYS protein was detected in the wild-type myotubes (CTL+), and a truncated DYS protein (about 400 kDa) was detected in the CinDel-corrected DMD myotubes (CinDel).