Figure 6: Expression of restored human dystrophin in vivo following cell transplantation. | Nature Communications

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

From: Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy

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 Fig. 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 fibres 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 fibres positive for human dystrophin. Scale bars indicate 100 μm. ND, not determined.

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