Research Progress on Disease Models Based on Induced Pluripotent Stem Cells

Dear Colleagues,

Induced pluripotent stem cell (iPSC) technology continues to redefine the possibilities of disease modeling and development of therapeutic approaches. By enabling the creation of patient-specific cell types capable of demonstrating human pathophysiology accurately, iPSCs have become a basis for investigating disease mechanisms, identifying therapeutic targets, and advancing precision medicine. Recent innovations—such as high-throughput multi-omics profiling, advanced 3D culture platforms, and organ-on-chip systems—are further expanding the power of iPSC-derived models. In addition, iPSC-based technology has entered a new era with its growing role in cell therapy development. iPSC-derived cell products, including cardiomyocytes, neural cells, immune cells, and epithelial tissues, are rapidly progressing toward clinical applications. These approaches offer unprecedented advantages, such as scalable production, standardized quality, and the potential to create universal donor cell lines.

With the advances of genome editing technologies such as the CRISPR system, the application of iPSC platforms is widely extended and the underlying mechanisms of abundant diseases are revealed at cellular level. Genetically modified iPSCs enable us to precisely correct pathogenic variants, establish isogenic controls, and engineer enhanced therapeutic cells with improved safety, efficacy, and immune compatibility. These advances are accelerating both discovery of disease mechanism and the development of next-generation therapies for non-curable diseases.

This Special Issue, “Research Progress on Disease Models Based on Induced Pluripotent Stem Cells,” invites contributions that highlight breakthrough technologies, new biological insights, and translational advances across the iPSC field.

Topics of interest include, but are not limited to, the following:

• Innovations in iPSC differentiation, maturation, and lineage engineering;
• Study on iPSC-based models of monogenic, complicated, or rare diseases;
• Disease mechanism studies using genome-edited or isogenic iPSC lines;
• Application of multi-omics technologies in iPSC disease modeling;
• Advances in iPSC-derived organoids and cell sheets;
• Development and clinical translation of iPSC-derived cell therapies;
• Engineering and application of gene-edited iPSCs for therapeutics of non-curable disorders;
• Strategies to generate iPSC-derived cell products for treatment of diseases;
• Precision medicine approaches leveraging patient-specific iPSC models.

Through this Special Issue, we aim to highlight the current development of the rapidly evolving spectrum of iPSC research—from fundamental mechanisms to therapeutic innovations—and to highlight how iPSC-based technologies are reshaping both biomedical science and clinical development.

Dr. Delger Bayarsaikhan
Guest Editor

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