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

Figure 1

Schematic representation of the Idua^-/- iPSC derivation and gene correction. Idua^-/- mouse embryonic fibroblasts were reprogrammed with STEMCCA lentivirus. The disrupting neomycin resistance gene (Neo^r) was removed, and the Idua gene was corrected with CRISPR/Cas9 gene editing technology.