ArticleBlood2012
Zinc-finger nuclease-mediated correction of α-thalassemia in iPS cells.
Article in Blood, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 47 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.
The trial behind it
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
47 citing papers in PubMed, 84 citations in OpenAlex.
- Trial
- Review
- Review
- Review
- Differentiation of human induced pluripotent stem cells into erythroid cells.Stem cell research & therapy · 2020Review
- A Small Key for a Heavy Door: Genetic Therapies for the Treatment of Hemoglobinopathies.Frontiers in genome editing · 2020Review
- CRISPR/Cas9 gene correction of HbH-CS thalassemia-induced pluripotent stem cells.Annals of hematology · 2019Article
- Modeling blood diseases with human induced pluripotent stem cells.Disease models & mechanisms · 2019Review
- Two decades of embryonic stem cells: a historical overview.Human reproduction open · 2019Article
- Gene editing of PKLR gene in human hematopoietic progenitors through 5' and 3' UTR modified TALEN mRNA.PloS one · 2019Article
- Recent Updates on Induced Pluripotent Stem Cells in Hematological Disorders.Stem cells international · 2019Review
- Interleukin-22 promotes phagolysosomal fusion to induce protection againstProceedings of the National Academy of Sciences of the United States of America · 2018Article
- Erythropoiesis: insights into pathophysiology and treatments in 2017.Molecular medicine (Cambridge, Mass.) · 2018Review
- Inside out: regenerative medicine for recessive dystrophic epidermolysis bullosa.Pediatric research · 2018Review
- Enhancement of β-Globin Gene Expression in Thalassemic IVS2-654 Induced Pluripotent Stem Cell-Derived Erythroid Cells by Modified U7 snRNA.Stem cells translational medicine · 2017Article
- Induced Pluripotent Stem Cells: Global Research Trends.BioResearch open access · 2017Review
- Article
- Distinct gene expression program dynamics during erythropoiesis from human induced pluripotent stem cells compared with adult and cord blood progenitors.BMC genomics · 2016Article
- hiPSC-derived iMSCs: NextGen MSCs as an advanced therapeutically active cell resource for regenerative medicine.Journal of cellular and molecular medicine · 2016Review
- Site-Specific Genome Engineering in Human Pluripotent Stem Cells.International journal of molecular sciences · 2016Review
Corrections and comments
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Authors and funding
2 authors at 1 institution in 1 country.
Funding
Abstract
Induced pluripotent stem (iPS) cell technology holds vast promises for a cure to the hemoglobinopathies. Constructs and methods to safely insert therapeutic genes to correct the genetic defect need to be developed. Site-specific insertion is a very attractive method for gene therapy because the risks of insertional mutagenesis are eliminated provided that a "safe harbor" is identified, and because a single set of validated constructs can be used to correct a large variety of mutations simplifying eventual clinical use. We report here the correction of α-thalassemia major hydrops fetalis in transgene-free iPS cells using zinc finger-mediated insertion of a globin transgene in the AAVS1 site on human chromosome 19. Homozygous insertion of the best of the 4 constructs tested led to complete correction of globin chain imbalance in erythroid cells differentiated from the corrected iPS cells.
Indexed as
Identifiers
What OpenQuestion holds
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.