ArticleStem cells translational medicine2016
Naïve Induced Pluripotent Stem Cells Generated From β-Thalassemia Fibroblasts Allow Efficient Gene Correction With CRISPR/Cas9.
Article in Stem cells translational medicine, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 36 papers.
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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
36 citing papers in PubMed.
- "Stem Cells in Regenerative Medicine: from Discovery and Molecular Mechanisms To Therapeutic Applications and Clinical Challenges".Cell biochemistry and biophysics · 2026Review
- "Stem Cells in Regenerative Medicine: from Discovery and Molecular Mechanisms To Therapeutic Applications and Clinical Challenges".Cell biochemistry and biophysics · 2026Review
- Therapeutic gene correction of HBB frameshift CD41-42 (-TCTT) deletion in human hematopoietic stem cells.Advanced biotechnology · 2025Article
- PRODH safeguards human naive pluripotency by limiting mitochondrial oxidative phosphorylation and reactive oxygen species production.EMBO reports · 2024Article
- Recent Research Trends in Stem Cells Using CRISPR/Cas-Based Genome Editing Methods.International journal of stem cells · 2024Review
- Gene editing and reprogramming of human fibroblast cells (hFBs) to human pluripotent stem cells (hiPSCs).Neuromethods · 2024Article
- Discordance between chromatin accessibility and transcriptional activity during the human primed-to-naïve pluripotency transition process.Cell regeneration (London, England) · 2023Article
- Female naïve human pluripotent stem cells carry X chromosomes with Xa-like and Xi-like folding conformations.Science advances · 2023Article
- Cell fate roadmap of human primed-to-naive transition reveals preimplantation cell lineage signatures.Nature communications · 2022Article
- Review
- Loss of neurodevelopmental-associated miR-592 impairs neurogenesis and causes social interaction deficits.Cell death & disease · 2022Article
- CRISPR Guide RNA Library Screens in Human Induced Pluripotent Stem Cells.Methods in molecular biology (Clifton, N.J.) · 2022Article
- Review
- Understanding the Potential of Genome Editing in Parkinson's Disease.International journal of molecular sciences · 2021Review
- Review
- Advances in Pluripotent Stem Cells: History, Mechanisms, Technologies, and Applications.Stem cell reviews and reports · 2020Review
- Review
- Long noncoding RNA CCDC144NL-AS1 knockdown induces naïve-like state conversion of human pluripotent stem cells.Stem cell research & therapy · 2019Article
- CRISP Points on EstablishingIndian journal of hematology & blood transfusion : an official journal of Indian Society of Hematology and Blood Transfusion · 2019Review
- Rare Opportunities: CRISPR/Cas-Based Therapy Development for Rare Genetic Diseases.Molecular diagnosis & therapy · 2019Review
Corrections and comments
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- Erratum issued
Authors and funding
12 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
unlabelledConventional primed human embryonic stem cells and induced pluripotent stem cells (iPSCs) exhibit molecular and biological characteristics distinct from pluripotent stem cells in the naïve state. Although naïve pluripotent stem cells show much higher levels of self-renewal ability and multidifferentiation capacity, it is unknown whether naïve iPSCs can be generated directly from patient somatic cells and will be superior to primed iPSCs. In the present study, we used an established 5i/L/FA system to directly reprogram fibroblasts of a patient with β-thalassemia into transgene-free naïve iPSCs with molecular signatures of ground-state pluripotency. Furthermore, these naïve iPSCs can efficiently produce cross-species chimeras. Importantly, using the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 nuclease genome editing system, these naïve iPSCs exhibit significantly improved gene-correction efficiencies compared with the corresponding primed iPSCs. Furthermore, human naïve iPSCs could be directly generated from noninvasively collected urinary cells, which are easily acquired and thus represent an excellent cell resource for further clinical trials. Therefore, our findings demonstrate the feasibility and superiority of using patient-specific iPSCs in the naïve state for disease modeling, gene editing, and future clinical therapy. SIGNIFICANCE: In the present study, transgene-free naïve induced pluripotent stem cells (iPSCs) directly converted from the fibroblasts of a patient with β-thalassemia in a defined culture system were generated. These naïve iPSCs, which show ground-state pluripotency, exhibited significantly improved single-cell cloning ability, recovery capacity, and gene-targeting efficiency compared with conventional primed iPSCs. These results provide an improved strategy for personalized treatment of genetic diseases such as β-thalassemia.
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