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. Author manuscript; available in PMC: 2015 Aug 18.
Published in final edited form as: Nat Commun. 2015 Feb 18;6:6244. doi: 10.1038/ncomms7244

Figure 6. Expression of restored human dystrophin in vivo following cell transplantation.

Figure 6

Human Δ48–50 DMD myoblasts were treated with SpCas9, CR1, and CR5 to delete exon 51 and sorted for GFP expression as shown in Figure 2. These sorted cells and untreated control cells were injected into the hind limbs of immunodeficient mice and assessed for human-specific protein expression in muscle fibers after 4 weeks post-transplantation. Cryosections were stained with anti-human spectrin, which is expressed by both uncorrected and corrected myoblasts that have fused into mouse myofibers, or anti-human dystrophin antibodies as indicated. White arrows indicate muscle fibers positive for human dystrophin. Scale bars indicate 100 μm.