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Editorial: Studying rare diseases using induced pluripotent stem cell (iPSC)-based model systems
by
Chen, Guokai
, Kiris, Erkan
, Francis, Kevin R.
in
Cell therapy
/ Cysts
/ disease modelling
/ Drug development
/ drug discovery
/ Editing
/ Generative artificial intelligence
/ Grants
/ induced pluripotent stem cells
/ Mutation
/ Organoids
/ Pathogenesis
/ Phenotypes
/ Photoreceptors
/ Pluripotency
/ R&D
/ Rare diseases
/ Regenerative medicine
/ Research & development
/ Stem cells
/ Toxicity
/ toxicity assays
/ Writing
2025
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Editorial: Studying rare diseases using induced pluripotent stem cell (iPSC)-based model systems
by
Chen, Guokai
, Kiris, Erkan
, Francis, Kevin R.
in
Cell therapy
/ Cysts
/ disease modelling
/ Drug development
/ drug discovery
/ Editing
/ Generative artificial intelligence
/ Grants
/ induced pluripotent stem cells
/ Mutation
/ Organoids
/ Pathogenesis
/ Phenotypes
/ Photoreceptors
/ Pluripotency
/ R&D
/ Rare diseases
/ Regenerative medicine
/ Research & development
/ Stem cells
/ Toxicity
/ toxicity assays
/ Writing
2025
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Do you wish to request the book?
Editorial: Studying rare diseases using induced pluripotent stem cell (iPSC)-based model systems
by
Chen, Guokai
, Kiris, Erkan
, Francis, Kevin R.
in
Cell therapy
/ Cysts
/ disease modelling
/ Drug development
/ drug discovery
/ Editing
/ Generative artificial intelligence
/ Grants
/ induced pluripotent stem cells
/ Mutation
/ Organoids
/ Pathogenesis
/ Phenotypes
/ Photoreceptors
/ Pluripotency
/ R&D
/ Rare diseases
/ Regenerative medicine
/ Research & development
/ Stem cells
/ Toxicity
/ toxicity assays
/ Writing
2025
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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).
Publisher
Frontiers Media SA,Frontiers Media S.A
Subject
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