Modeling rare genetic disease with patient-derived induced pluripotent stem cells: reassessment of the minimum numbers of lines needed
Published in Stem Cells Translational Medicine, 2025
Recommended citation: Dinasarapu AR , Sutcliffe DJ, Grychowski L, Ozel E, Thite A, Visser JE, Hess EJ, Kolk SM, Jinnah HA, Modeling rare genetic disease with patient-derived induced pluripotent stem cells: reassessment of the minimum numbers of lines needed, Stem Cells Translational Medicine, 14, 8, 2025. https://doi.org/10.1093/stcltm/szaf032
Induced pluripotent stem cells (iPSCs) are widely used to model human genetic diseases. The most common strategy involves collecting cells from relevant individuals and then reprogramming them into iPSCs. This strategy is very powerful, but finding enough individuals with a specific genetic disease can be challenging, especially since most are rare.