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

Figure 2

Idua^-/- mouse iPSC derivation and validation. (a) Phase contrast image of miPSC colonies. (b) Stem cell marker gene expression in three different miPSC lines. Mouse embryonic stem cell (mES) as control. (c) Immunofluorescent staining for stem cell markers, SSEA-1 and Oct4. Nuclear counter staining with DAPI. (d) Immunofluorescent staining demonstrated lineage-specific marker protein-positive cells. (e) Biochemical analyses demonstrate IDUA activity in wild-type (WT) and IDUA knockout (IDUA KO) fibroblasts and iPSCs derived from each cell line.

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