ArticleExperimental & molecular medicine2012
Disease-specific induced pluripotent stem cells: a platform for human disease modeling and drug discovery.
Article in Experimental & molecular medicine, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 53 papers.
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Who cites it
53 citing papers in PubMed.
- Generation of marmoset monkeys with a non-mosaic disruption of the OTOF gene as a model of human deafness.Nature communications · 2026Article
- Effect of Octacalcium Phosphate on Osteogenic Differentiation of Induced Pluripotent Stem Cells in a 3D Hybrid Spheroid Culture.Biomimetics (Basel, Switzerland) · 2025Article
- Magnetically reshapable 3D multi-electrode arrays of liquid metals for electrophysiological analysis of brain organoids.Nature communications · 2025Article
- Clinical translation of human iPSC technologies: advances, safety concerns, and future directions.Frontiers in cell and developmental biology · 2025Review
- Bottom-up Biomaterial strategies for creating tailored stem cells in regenerative medicine.Frontiers in bioengineering and biotechnology · 2025Review
- Neuronal Cell Differentiation of iPSCs for the Clinical Treatment of Neurological Diseases.Biomedicines · 2024Review
- Research Models to Study Ferroptosis's Impact in Neurodegenerative Diseases.Pharmaceutics · 2023Review
- Semi-automated optimized method to isolate CRISPR/Cas9 edited human pluripotent stem cell clones.Stem cell research & therapy · 2023Article
- Transplantation of PSA-NCAM-Positive Neural Precursors from Human Embryonic Stem Cells Promotes Functional Recovery in an Animal Model of Spinal Cord Injury.Tissue engineering and regenerative medicine · 2022Article
- Passage number affects differentiation of sensory neurons from human induced pluripotent stem cells.Scientific reports · 2022Article
- Stem Cell Transplantation Therapy and Neurological Disorders: Current Status and Future Perspectives.Biology · 2022Review
- Recapitulating pancreatic cell-cell interactions through bioengineering approaches: the momentous role of non-epithelial cells for diabetes cell therapy.Cellular and molecular life sciences : CMLS · 2021Review
- Recent progress in pancreatic islet cell therapy.Inflammation and regeneration · 2021Review
- Targeted Integration of Inducible Caspase-9 in Human iPSCs Allows EfficientInternational journal of molecular sciences · 2020Article
- 3,2'-Dihydroxyflavone Improves the Proliferation and Survival of Human Pluripotent Stem Cells and Their Differentiation into Hematopoietic Progenitor Cells.Journal of clinical medicine · 2020Article
- Expression of Normal or Mutated X-LinkedJournal of dental research · 2020Article
- Review
- A compendious summary of Parkinson's disease patient-derived iPSCs in the first decade.Annals of translational medicine · 2019Review
- Modelling mitochondrial dysfunction in Alzheimer's disease using human induced pluripotent stem cells.World journal of stem cells · 2019Review
- Important advances in Alzheimer's disease from the use of induced pluripotent stem cells.Journal of biomedical science · 2019Review
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Authors and funding
12 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The generation of disease-specific induced pluripotent stem cell (iPSC) lines from patients with incurable diseases is a promising approach for studying disease mechanisms and drug screening. Such innovation enables to obtain autologous cell sources in regenerative medicine. Herein, we report the generation and characterization of iPSCs from fibroblasts of patients with sporadic or familial diseases, including Parkinson's disease (PD), Alzheimer's disease (AD), juvenile-onset, type I diabetes mellitus (JDM), and Duchenne type muscular dystrophy (DMD), as well as from normal human fibroblasts (WT). As an example to modeling disease using disease-specific iPSCs, we also discuss the previously established childhood cerebral adrenoleukodystrophy (CCALD)- and adrenomyeloneuropathy (AMN)-iPSCs by our group. Through DNA fingerprinting analysis, the origins of generated disease-specific iPSC lines were identified. Each iPSC line exhibited an intense alkaline phosphatase activity, expression of pluripotent markers, and the potential to differentiate into all three embryonic germ layers: the ectoderm, endoderm, and mesoderm. Expression of endogenous pluripotent markers and downregulation of retrovirus-delivered transgenes [OCT4 (POU5F1), SOX2, KLF4, and c-MYC] were observed in the generated iPSCs. Collectively, our results demonstrated that disease-specific iPSC lines characteristically resembled hESC lines. Furthermore, we were able to differentiate PD-iPSCs, one of the disease-specific-iPSC lines we generated, into dopaminergic (DA) neurons, the cell type mostly affected by PD. These PD-specific DA neurons along with other examples of cell models derived from disease-specific iPSCs would provide a powerful platform for examining the pathophysiology of relevant diseases at the cellular and molecular levels and for developing new drugs and therapeutic regimens.
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