ArticleMolecular therapy. Methods & clinical development2022
Generation of hypoimmunogenic induced pluripotent stem cells by CRISPR-Cas9 system and detailed evaluation for clinical application.
Article in Molecular therapy. Methods & clinical development, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 40 papers.
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Who cites it
40 citing papers in PubMed, 76 citations in OpenAlex.
- Genetically engineered hypoimmunogenic iPSCs: Strategies and considerations for clinical applications.Regenerative therapy · 2026Review
- First evidence of the circulation of induced pluripotent stem cell-derived platelets in humans.Stem cells translational medicine · 2026Article
- Advances in the differentiation of induced pluripotent stem cells into vascular cells for the treatment of diabetic microvascular disease.Cardiovascular diabetology · 2026Review
- Engineering Immune Cell to Counteract Aging and Aging-Associated Diseases.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Review
- Reversal of ACLF and ALF using whole blood extracorporeal system combining HLA-depleted liver organoids with granulocyte-monocyte apheresis.Journal of hepatology · 2026Article
- Intrathecal CRISPR-edited allogeneic IL-13Rα2 CAR T Cells for recurrent high-grade Glioma: preclinical characterization and phase I trial.Nature communications · 2026Article
- Gene Editing of Pluripotent Stem Cell-Derived Hepatic Cells for Liver Disease Modeling and Therapeutic Development.Biomolecules & therapeutics · 2026Review
- The precision strategy of human genome correction via a set of circular donor DNA and its cleaver.Frontiers in genome editing · 2026Article
- Extending the residence period of allogeneic iPSC-derived invariant natural killer T cells in humanized mice by editing HLA class I expression.Frontiers in cell and developmental biology · 2026Article
- Unveiling genomic rearrangements in engineered iPSC lines by optical genome mapping.Molecular therapy. Methods & clinical development · 2025Article
- Scaffold-free cell sheet therapies: clinical advances, global approval landscapes, and strategic directions to address regenerative medicine barriers.Stem cell research & therapy · 2025Review
- Hypoimmune stem cells and islets: hype or a true breakthrough in diabetes treatment?Cellular & molecular biology letters · 2025Review
- Stem Cell Therapy in Ischemic Heart Failure.American journal of cardiovascular drugs : drugs, devices, and other interventions · 2025Review
- The effects and mechanisms of stem cells and exosomes on neurological function protection and recovery after ischemic stroke.Neuroprotection (Chichester, England) · 2025Review
- Pluripotent stem cell-derived cardiomyocyte transplantation: marching from bench to bedside.Science China. Life sciences · 2025Review
- Engineering the next generation of allogeneic CAR cells: iPSCs as a scalable and editable platform.Stem cell reports · 2025Review
- Article
- Managing allorejection in off-the-shelf CAR-engineered cell therapies.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Advantages and challenges of using allogeneic vs. autologous sources for neuronal cell replacement in Parkinson's disease: Insights from non-human primate studies.Brain research bulletin · 2025Review
- Modelling human brain development and disease with organoids.Nature reviews. Molecular cell biology · 2025Review
Corrections and comments
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Authors and funding
13 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
In order to expand the promise of regenerative medicine using allogeneic induced pluripotent stem cells (iPSCs), precise and efficient genome editing of human leukocyte antigen (HLA) genes would be advantageous to minimize the immune rejection caused by mismatches of HLA type. However, clinical-grade genome editing of multiple HLA genes in human iPSC lines remains unexplored. Here, we optimized the protocol for good manufacturing practice (GMP)-compatible CRISPR-Cas9 genome editing to deplete the three gene locus (HLA-A, HLA-B, and CIITA genes) simultaneously in HLA homozygous iPSCs. The use of HLA homozygous iPSCs has one main advantage over heterozygous iPSCs for inducing biallelic knockout by a single gRNA. RNA-seq and flow cytometry analyses confirmed the successful depletion of HLAs, and lineage-specific differentiation into cardiomyocytes was verified. We also confirmed that the pluripotency of genome-edited iPSCs was successfully maintained by the three germ layers of differentiation. Moreover, whole-genome sequencing, karyotyping, and optical genome mapping analyses revealed no evident genomic abnormalities detected in some clones, whereas unexpected copy number losses, chromosomal translocations, and complex genomic rearrangements were observed in other clones. Our results indicate the importance of multidimensional analyses to ensure the safety and quality of the genome-edited cells. The manufacturing and assessment pipelines presented here will be the basis for clinical-grade genome editing of iPSCs.
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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.