ArticleStem cell reports2016
Gene Correction of iPSCs from a Wiskott-Aldrich Syndrome Patient Normalizes the Lymphoid Developmental and Functional Defects.
Article in Stem cell reports, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 34 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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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
34 citing papers in PubMed, 46 citations in OpenAlex.
- Advancements in gene therapy for Wiskott-Aldrich syndrome: from early trials to emerging approaches.International journal of hematology · 2026Review
- Gene Correction of Wiskott-Aldrich syndrome iPS Cells Rescues Proplatelet Defects and Improves Platelet Size.Thrombosis and haemostasis · 2025Article
- [Advances in gene therapy for inborn errors of immunity].Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics · 2024Review
- Review
- Gene editing-based targeted integration for correction of Wiskott-Aldrich syndrome.Molecular therapy. Methods & clinical development · 2024Article
- Correcting inborn errors of immunity: From viral mediated gene addition to gene editing.Seminars in immunology · 2023Review
- The Wiskott-Aldrich syndrome protein is required for positive selection during T-cell lineage differentiation.Frontiers in immunology · 2023Article
- Review
- Wiskott-Aldrich syndrome protein forms nuclear condensates and regulates alternative splicing.Nature communications · 2022Article
- Review
- Genome editing for primary immunodeficiencies: A therapeutic perspective on Wiskott-Aldrich syndrome.Frontiers in immunology · 2022Review
- Review
- Review
- Review
- [Gene editing for the treatment of primary immunodeficiency disease].Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics · 2021Review
- Review
- Natural Killer Cells Generated From Human Induced Pluripotent Stem Cells Mature to CD56Frontiers in immunology · 2021Article
- Gene Editing for the Treatment of Primary Immunodeficiency Diseases.Human gene therapy · 2021Review
- Differentiation of human induced pluripotent stem cells into erythroid cells.Stem cell research & therapy · 2020Review
- Treating primary immunodeficiencies with defects in NK cells: from stem cell therapy to gene editing.Stem cell research & therapy · 2020Review
Corrections and comments
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Authors and funding
17 authors at 5 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Wiskott-Aldrich syndrome (WAS) is an X-linked primary immunodeficiency disease caused by mutations in the gene encoding the WAS protein (WASp). Here, induced pluripotent stem cells (iPSCs) were derived from a WAS patient (WAS-iPSC) and the endogenous chromosomal WAS locus was targeted with a wtWAS-2A-eGFP transgene using zinc finger nucleases (ZFNs) to generate corrected WAS-iPSC (cWAS-iPSC). WASp and GFP were first expressed in the earliest CD34(+)CD43(+)CD45(-) hematopoietic precursor cells and later in all hematopoietic lineages examined. Whereas differentiation to non-lymphoid lineages was readily obtained from WAS-iPSCs, in vitro T lymphopoiesis from WAS-iPSC was deficient with few CD4(+)CD8(+) double-positive and mature CD3(+) T cells obtained. T cell differentiation was restored for cWAS-iPSCs. Similarly, defects in natural killer cell differentiation and function were restored on targeted correction of the WAS locus. These results demonstrate that the defects exhibited by WAS-iPSC-derived lymphoid cells were fully corrected and suggests the potential therapeutic use of gene-corrected WAS-iPSCs.
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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.