CRISPR/Cas9 Genome Editing in LGMD2A/R1 Patient-Derived Induced Pluripotent Stem and Skeletal Muscle Progenitor Cells

Figure 1

(a) Induced pluripotent stem cells and genome editing to model genetic correction of limb-girdle muscular dystrophy (LGMD2A). CRISPR/Cas9 mediated homology-directed repair (HDR) at the calpain-3 (CAPN3) locus on the level of human LGMD2A iPS cells and subsequent differentiation to wildtype (wt) skeletal muscle cells (upper panel). CRISPR/Cas9 mediated HDR at the CAPN3 locus on the level of human iPS-derived CD82+ skeletal muscle progenitor cells (lower panel). (b) Strategy to perform gene correction of LGMD 2A patient hiPS cell line: cartoon illustrating the targeting strategy followed for generating the isogenic line using CRISPR/Cas9 genome editing.

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