ArticleiScience2019
Using Gene Editing to Establish a Safeguard System for Pluripotent Stem-Cell-Based Therapies.
Article in iScience, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.
What it found
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
15 citing papers in PubMed, 23 citations in OpenAlex.
- In vivo generation of CAR macrophages via the enucleated mesenchymal stem cell delivery system for glioblastoma therapy.Proceedings of the National Academy of Sciences of the United States of America · 2025Article
- The Use of MSCs, iPSCs, and EVs in the Repair of Human MSK Tissues: Is Ultimate Success Dependent on Developing Excellent Implant Materials as Well as Creating an Optimal Environment for Implantation? What Is the Rationale for These Choices?International journal of molecular sciences · 2025Review
- Importance of multiple endocrine cell types in islet organoids for type 1 diabetes treatment.Translational research : the journal of laboratory and clinical medicine · 2022Review
- Scalable manufacturing of clinical-grade differentiated cardiomyocytes derived from human-induced pluripotent stem cells for regenerative therapy.Cell proliferation · 2022Review
- Islet organoid as a promising model for diabetes.Protein & cell · 2022Review
- Inducible caspase-9 suicide gene under control of endogenous oct4 to safeguard mouse and human pluripotent stem cell therapy.Molecular therapy. Methods & clinical development · 2022Article
- Tumorigenicity risk of iPSCsPrecision clinical medicine · 2022Review
- Strategies to Improve the Safety of iPSC-Derived β Cells for β Cell Replacement in Diabetes.Transplant international : official journal of the European Society for Organ Transplantation · 2022Review
- CAR-NK cells from engineered pluripotent stem cells: Off-the-shelf therapeutics for all patients.Stem cells translational medicine · 2021Article
- EditingInternational journal of molecular sciences · 2021Review
- Exploiting B Cell Transfer for Cancer Therapy: Engineered B Cells to Eradicate Tumors.International journal of molecular sciences · 2021Review
- Improved safety of induced pluripotent stem cell-derived antigen-presenting cell-based cancer immunotherapy.Molecular therapy. Methods & clinical development · 2021Article
- Salicylic diamines selectively eliminate residual undifferentiated cells from pluripotent stem cell-derived cardiomyocyte preparations.Scientific reports · 2021Article
- Stem Cell-Based Clinical Trials for Diabetes Mellitus.Frontiers in endocrinology · 2021Review
- Improving the safety of human pluripotent stem cell therapies using genome-edited orthogonal safeguards.Nature communications · 2020Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 3 institutions in 2 countries.
Funding
Abstract
A major challenge in using human pluripotent stem cells (hPSCs) in therapy is the risk of teratoma formation due to contaminating undifferentiated stem cells. We used CRISPR-Cas9 for in-frame insertion of a suicide gene, iC9, into the endogenous SOX2 locus in human embryonic stem cell (ESC) line H1 for specific eradication of undifferentiated cells without affecting differentiated cells. This locus was chosen over NANOG and OCT4, two other well-characterized stem cell loci, due to significantly reduced off-target effect. We showed that undifferentiated H1-iC9 cells were induced to apoptosis by iC9 inducer AP1903, whereas differentiated cell lineages including hematopoietic cells, neurons, and islet beta-like cells were not affected. We also showed that AP1903 selectively removed undifferentiated H1-iC9 cells from a mixed cell population. This strategy therefore provides a layer of safety control before transplantation of a stem-cell-derived product in therapy.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.