Figure 8.

In vivo transplantation of PHSats into immune- and calpain 3-deficient mice

(A) Representative image of immunofluorescence staining against human Lamin A/C and human Spectrin labeling donor nuclei and donor-derived human muscle fibers in injected TA muscles. Top: grafted muscle from edited cells. Bottom: graft formed by unedited cells. The right image depicts a close-up of the area of the transplanted muscle. Scale bars, 50 μM. (B) Quantification of engrafted myofibers. Spectrin-positive myofibers were counted. Grafts from edited cells are slightly bigger; p > 0.08, n = 7 animals; each quantification was done from a separate mouse. (C) Detection of CAPN3 expression by RT-qPCR in grafted muscles. Control is C56BL/6; treated and untreated are C3KO-NSG. CAPN3 expression is recovered after transplantation of gene edited PHSats. Relative expression was calculated to GAPDH. Statistics were done using one-way ANOVA; p < 0.01, n = 3.