ArticleScience advances2022
Development of iPSC-based clinical trial selection platform for patients with ultrarare diseases.
Article in Science advances, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.
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Who cites it
15 citing papers in PubMed.
- Metabolic crisis and TRPM4 activation cause QT prolongation in TANGO2 deficiency disorder.Cardiovascular research · 2026Article
- Germanane Quantum Dots Promote Metabolic Reprogramming of Immune Cells Toward Regulatory T Cells and Suppress Inflammation In Vitro and In Vivo.Advanced functional materials · 2026Article
- TCA cycle intermediates as an adjunct strategy for human iPSC-derived cardiomyocyte maturation.BBA advances · 2026Article
- Moyamoya disease: epidemiology, clinical features, pathogenesis, diagnosis and therapeutic interventions.Molecular biomedicine · 2025Review
- Evolution of the management of ultrarare inherited arrhythmias and cardiomyopathies.Future cardiology · 2025Article
- Metabolic profiles and potential antioxidant mechanisms of hawk tea.Scientific reports · 2025Article
- Exploiting induced pluripotent stem cell-derived retinal pigment epithelium to unravel host-pathogen interaction in ocular tuberculosis: a reverse translationalFrontiers in ophthalmology · 2025Article
- Preclinical biodistribution and safety evaluation of human iPSC-derived dopaminergic neural progenitor cells for Parkinson's disease.Frontiers in cell and developmental biology · 2025Article
- Clinical trials in-a-dish for cardiovascular medicine.European heart journal · 2024Review
- iPSC-Based Disease Modeling and Functional Assessment of Neurons in Patients with Metabolic Disorder.Methods in molecular biology (Clifton, N.J.) · 2024Article
- Exploring the promising potential of induced pluripotent stem cells in cancer research and therapy.Molecular cancer · 2023Review
- Modified ECM-Based Bioink for 3D Printing of Multi-Scale Vascular Networks.Gels (Basel, Switzerland) · 2023Article
- Engineering considerations of iPSC-based personalized medicine.Biomaterials research · 2023Review
- Bridging the translational gap: what can synaptopathies tell us about autism?Frontiers in molecular neuroscience · 2023Review
- Towards establishing human body-on-a-chip systems.Stem cell research & therapy · 2022Article
Corrections and comments
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Authors and funding
12 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
A "Leap-of-Faith" approach is used to treat patients with previously unknown ultrarare pathogenic mutations, often based on evidence from patients having dissimilar but more prevalent mutations. This uncertainty reflects the need to develop personalized prescreening platforms for these patients to assess drug efficacy before considering clinical trial enrollment. In this study, we report an 18-year-old patient with ultrarare Leigh-like syndrome. This patient had previously participated in two clinical trials with unfavorable responses. We established an induced pluripotent stem cell (iPSC)-based platform for this patient, and assessed the efficacy of a panel of drugs. The iPSC platform validated the safety and efficacy of the screened drugs. The efficacy of three of the screened drugs was also investigated in the patient. After 3 years of treatment, the drugs were effective in shifting the metabolic profile of this patient toward healthy control. Therefore, this personalized iPSC-based platform can act as a prescreening tool to help in decision-making with respect to patient's participation in future clinical trials.
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