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Editorial: Studying rare diseases using induced pluripotent stem cell (iPSC)-based model systems
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Editorial: Studying rare diseases using induced pluripotent stem cell (iPSC)-based model systems
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Editorial: Studying rare diseases using induced pluripotent stem cell (iPSC)-based model systems
Editorial: Studying rare diseases using induced pluripotent stem cell (iPSC)-based model systems
Journal Article

Editorial: Studying rare diseases using induced pluripotent stem cell (iPSC)-based model systems

2025
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Overview
To facilitate relevant scientific developments, it is critical that human cells, especially cell types affected in specific rare diseases, be obtained on a large scale for scientific work. [...]Parvatam et al. discussed complex in vitro models (CIVM), including iPSCs, organoids, and organs-on-chip models, as powerful tools for developing effective therapies with a higher clinical translation chance (Parvatam et al.). Developing methods for the precise induction of cellular complexes and functional tissues would enable more accurate disease modeling. [...]although not covered in this Research Topic, iPSC-based cell replacement therapies could be a viable option for the treatment of specific cellular phenotypes in rare diseases, considering the potential of iPSC-based cell therapies in regenerative medicine (Takahashi, 2025).