Induced Pluripotent Stem Cell Derivation and Ex Vivo Gene Correction Using a Mucopolysaccharidosis Type 1 Disease Mouse Model

Figure 3

CRISPR gene editing and validations. (a) Schematic representation of CRISPR gene editing strategy. (b) Flow cytometric analysis of CRISPR/Cas9-OFP vector transfection efficiency. GFP channel was used to detect autofluorescence of dead cells. (c) Stem cell marker gene expression in two gene-edited miPSC lines. (d) Identification of three germ layers in teratomas. Red arrows indicate typical histological feature of each lineage. (e) Biochemical analyses to validate Idua enzyme function restoration. and . n.s: no significant difference using one-way ANOVA with Bonferroni post hoc analysis and Dunnett’s multiple comparison. (f) The retention of restored Idua enzyme activity was confirmed after inducing cell differentiation (Fib.: fibroblast-like cells, Ne./Sp.: spontaneously induced neuronal-shape cells). and . n.s: no significant difference using one-way ANOVA with Bonferroni post hoc analysis and Dunnett’s multiple comparison.

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