This review provides a guide to the conceptual and practical issues to consider when trying to generate an iPSc model that accurately recapitulates the features of a human genetic disease. The authors highlight recent successes in modeling genetic diseases using iPSCs and offers a perspective on the next steps that will be needed to improve current iPSC-based disease models.
- Gustavo Tiscornia
- Erica Lorenzo Vivas
- Juan Carlos Izpisúa Belmonte
