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Fig. 6 | Genome Medicine

Fig. 6

From: In vivo genome editing in mouse restores dystrophin expression in Duchenne muscular dystrophy patient muscle fibers

Fig. 6

In vivo differentiation of DMD–MDSCs into muscle fibers. a DMD–MDSCs infected with lentivirus-expressing luciferase were transplanted into recipients, and engraftment was monitored by imaging over a period of 4 weeks. Bioluminescence images of representative injected mice were acquired on the indicated days. b qPCR analysis of human mitochondrial DNA revealed the presence of human cells in PDX DMD mouse following injection of DMD–MDSCs over a period of 4 weeks. Mouse DNA samples (M) and human DNA samples (H), as well as a series of mouse–human cell dilution samples, were run as negative and positive controls to estimate the degree of human cell contribution. The red dotted lines indicated that the proportion of human cells in the TA muscle of PDX DMD was between 2 and 2.5%. c Immunofluorescence staining of laminin (green), human spectrin (red signal in sarcolemma), and human Lamin A+C (red signal in nucleus) in the TA muscle of PDX DMD mice; nuclei were counterstained with DAPI (blue). Scale bar, 100 μm

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