ArticleScientific reports2017
TALEN-mediated functional correction of human iPSC-derived macrophages in context of hereditary pulmonary alveolar proteinosis.
Article in Scientific reports, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.
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Who cites it
22 citing papers in PubMed, 30 citations in OpenAlex.
- Induced pluripotent stem cell-derived macrophages (iMacs) in translational medicine: Disease models, drug testing, and therapeutic applications.Molecular biology reports · 2026Review
- Induced pluripotent stem cell-derived macrophages as a platform for modelling human disease.Nature reviews. Immunology · 2025Review
- A dual-fluorescence assay for gene delivery vehicle screening in macrophages with an inflammation-inducible reporter construct.BMC methods · 2025Article
- Reporter Alleles in hiPSCs: Visual Cues on Development and Disease.International journal of molecular sciences · 2024Review
- A Model of iPSC-Derived Macrophages withInternational journal of molecular sciences · 2023Article
- Alveolar macrophages in pulmonary alveolar proteinosis: origin, function, and therapeutic strategies.Frontiers in immunology · 2023Review
- Human pluripotent stem cell-derived macrophages and macrophage-derived exosomes: therapeutic potential in pulmonary fibrosis.Stem cell research & therapy · 2022Review
- Macrophages derived from pluripotent stem cells: prospective applications and research gaps.Cell & bioscience · 2022Review
- Establishment and characterization of a novel human induced pluripotent stem cell line stably expressing the iRFP720 reporter.Scientific reports · 2022Article
- Review
- Pulmonary transplantation of alpha-1 antitrypsin (AAT)-transgenic macrophages provides a source of functional human AAT in vivo.Gene therapy · 2021Review
- A 3D iPSC-differentiation model identifies interleukin-3 as a regulator of early human hematopoietic specification.Haematologica · 2021Article
- Alveolar proteinosis of genetic origins.European respiratory review : an official journal of the European Respiratory Society · 2020Article
- Review
- Integration of nano- and biotechnology for beta-cell and islet transplantation in type-1 diabetes treatment.Cell proliferation · 2020Review
- Targeted Integration of Inducible Caspase-9 in Human iPSCs Allows EfficientInternational journal of molecular sciences · 2020Article
- DNA repair fidelity in stem cell maintenance, health, and disease.Biochimica et biophysica acta. Molecular basis of disease · 2020Review
- Macrophages Derived From Human Induced Pluripotent Stem Cells Are Low-Activated "Naïve-Like" Cells Capable of Restricting Mycobacteria Growth.Frontiers in immunology · 2020Article
- Targeted Integration and High-Level Transgene Expression in AAVS1 Transgenic Mice after In Vivo HSC Transduction with HDAd5/35++ Vectors.Molecular therapy : the journal of the American Society of Gene Therapy · 2019Article
- Concise Review: Modeling Neurodegenerative Diseases with Human Pluripotent Stem Cell-Derived Microglia.Stem cells (Dayton, Ohio) · 2019Review
Corrections and comments
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Authors and funding
6 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Hereditary pulmonary alveolar proteinosis (herPAP) constitutes a rare, life threatening lung disease characterized by the inability of alveolar macrophages to clear the alveolar airspaces from surfactant phospholipids. On a molecular level, the disorder is defined by a defect in the CSF2RA gene coding for the GM-CSF receptor alpha-chain (CD116). As therapeutic options are limited, we currently pursue a cell and gene therapy approach aiming for the intrapulmonary transplantation of gene-corrected macrophages derived from herPAP-specific induced pluripotent stem cells (herPAP-iPSC) employing transcriptional activator-like effector nucleases (TALENs). Targeted insertion of a codon-optimized CSF2RA-cDNA driven by the hybrid cytomegalovirus (CMV) early enhancer/chicken beta actin (CAG) promoter into the AAVS1 locus resulted in robust expression of the CSF2RA gene in gene-edited herPAP-iPSCs as well as thereof derived macrophages. These macrophages displayed typical morphology, surface phenotype, phagocytic and secretory activity, as well as functional CSF2RA expression verified by STAT5 phosphorylation and GM-CSF uptake studies. Thus, our study provides a proof-of-concept, that TALEN-mediated integration of the CSF2RA gene into the AAVS1 safe harbor locus in patient-specific iPSCs represents an efficient strategy to generate functionally corrected monocytes/macrophages, which in the future may serve as a source for an autologous cell-based gene therapy for the treatment of herPAP.
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