ArticleMolecular therapy : the journal of the American Society of Gene Therapy2015
An AAVS1-targeted minigene platform for correction of iPSCs from all five types of chronic granulomatous disease.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 43 papers.
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Who cites it
43 citing papers in PubMed, 70 citations in OpenAlex.
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- Differentiation of human hyalocytes from induced pluripotent stem cells through ascorbic acid treatment.Human cell · 2025Article
- Optimizing approaches for targeted integration of transgenic cassettes by integrase-mediated cassette exchange in mouse and human stem cells.Stem cells (Dayton, Ohio) · 2025Article
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- Multidimensional Response Surface Methodology for the Development of a Gene Editing Protocol for p67Human gene therapy · 2024Article
- Self-organized yolk sac-like organoids allow for scalable generation of multipotent hematopoietic progenitor cells from induced pluripotent stem cells.Cell reports methods · 2023Article
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- Chronic Granulomatous Disease: a Comprehensive Review.Clinical reviews in allergy & immunology · 2021Review
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- Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair.Gene therapy · 2021Article
- Suppression of HIV-1 Integration by Targeting HIV-1 Integrase for Degradation with A Chimeric Ubiquitin Ligase.Virologica Sinica · 2021Article
- NADPH oxidase correction by mRNA transfection of apheresis granulocytes in chronic granulomatous disease.Blood advances · 2020Article
- Treating primary immunodeficiencies with defects in NK cells: from stem cell therapy to gene editing.Stem cell research & therapy · 2020Review
- CRISPR-Directed Therapeutic Correction at theMolecular therapy. Methods & clinical development · 2020Article
- Gene editing and CRISPR in the clinic: current and future perspectives.Bioscience reports · 2020Review
- Differential Transgene Silencing of Myeloid-Specific Promoters in theHuman gene therapy · 2020Article
- CRISPR/Cas9-modified hematopoietic stem cells-present and future perspectives for stem cell transplantation.Bone marrow transplantation · 2019Review
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Authors and funding
17 authors at 5 institutions in 1 country.
Funding
Abstract
There are five genetic forms of chronic granulomatous disease (CGD), resulting from mutations in any of five subunits of phagocyte oxidase, an enzyme complex in neutrophils, monocytes, and macrophages that produces microbicidal reactive oxygen species. We generated induced pluripotent stem cells (iPSCs) from peripheral blood CD34(+) hematopoietic stem cells of patients with each of five CGD genotypes. We used zinc finger nuclease (ZFN) targeting the AAVS1 safe harbor site together with CGD genotype-specific minigene plasmids with flanking AAVS1 sequence to target correction of iPSC representing each form of CGD. We achieved targeted insertion with constitutive expression of desired oxidase subunit in 70-80% of selected iPSC clones. Neutrophils and macrophages differentiated from corrected CGD iPSCs demonstrated restored oxidase activity and antimicrobial function against CGD bacterial pathogens Staphylococcus aureus and Granulibacter bethesdensis. Using a standard platform that combines iPSC generation from peripheral blood CD34(+) cells and ZFN mediated AAVS1 safe harbor minigene targeting, we demonstrate efficient generation of genetically corrected iPSCs using an identical approach for all five genetic forms of CGD. This safe harbor minigene targeting platform is broadly applicable to a wide range of inherited single gene metabolic disorders.
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