Figure 4. Proposed concept of designer nuclease-mediated correction of patient-specific iPSC for autologous transplantation.

Patient-specific iPSC can be generated from somatic cells (e.g. fibroblasts, blood cells) and reprogrammed into pluripotent stem cells as discussed in the text. Targeted gene correction can be mediated via either zinc-finger nucleases (ZFN), transcription activator like effector nucleases (TALEN) or RNA guided-nucleases of patient-derived cells either before or after reprogramming to iPSC. Disease-corrected somatic cells can be derived from the iPSC and reintroduced into the patient as an autologous transplant.