ArticleScientific reports2019
Effective CRISPR/Cas9-mediated correction of a Fanconi anemia defect by error-prone end joining or templated repair.
Article in Scientific reports, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.
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Who cites it
15 citing papers in PubMed, 29 citations in OpenAlex.
- Reactive Oxygen Species in Cardiovascular and Hematological Diseases: Mechanisms and Therapeutic Strategies.Cureus · 2026Review
- Integrating CRISPR/Cas technology with clinical trials: Principles, progress and challenges.Asian journal of pharmaceutical sciences · 2025Review
- Removal of TREX1 activity enhances CRISPR-Cas9-mediated homologous recombination.Nature biotechnology · 2025Article
- [Advancements in CRISPR-Cas9 for Fanconi anemia].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2025Review
- A review on molecular scissoring with CRISPR/Cas9 genome editing technology.Toxicology research · 2024Review
- Advances in CRISPR-Cas technology and its applications: revolutionising precision medicine.Frontiers in genome editing · 2024Review
- Advances in Gene Therapy Techniques to TreatBiomolecules · 2022Review
- The origin of unwanted editing byproducts in gene editing.Acta biochimica et biophysica Sinica · 2022Article
- [Research progress of Fanconi anemia and DNA interstrand crosslink repair].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2022Article
- Cell and Gene Therapy for Anemia: Hematopoietic Stem Cells and Gene Editing.International journal of molecular sciences · 2021Review
- der(1)t(1;1)(p36;q10) as a Rare Variant of 1q Gain in Fanconi Anemia-Associated Myelodysplastic Syndrome.Annals of laboratory medicine · 2021Article
- Gene Editing of Hematopoietic Stem Cells: Hopes and Hurdles Toward Clinical Translation.Frontiers in genome editing · 2021Review
- Current trends in gene recovery mediated by the CRISPR-Cas system.Experimental & molecular medicine · 2020Review
- Gene Correction of Point Mutations Using PolyPurine Reverse Hoogsteen Hairpins Technology.Frontiers in genome editing · 2020Review
- CRISPR/Cas system: An emerging technology in stem cell research.World journal of stem cells · 2019Review
Corrections and comments
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Authors and funding
11 authors at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Fanconi anemia (FA) is a cancer predisposition syndrome characterized by congenital abnormalities, bone marrow failure, and hypersensitivity to aldehydes and crosslinking agents. For FA patients, gene editing holds promise for therapeutic applications aimed at functionally restoring mutated genes in hematopoietic stem cells. However, intrinsic FA DNA repair defects may obstruct gene editing feasibility. Here, we report on the CRISPR/Cas9-mediated correction of a disruptive mutation in Fancf. Our experiments revealed that gene editing could effectively restore Fancf function via error-prone end joining resulting in a 27% increased survival in the presence of mitomycin C. In addition, templated gene correction could be achieved after double strand or single strand break formation. Although templated gene editing efficiencies were low (≤6%), FA corrected embryonic stem cells acquired a strong proliferative advantage over non-corrected cells, even without imposing genotoxic stress. Notably, Cas9 nickase activity resulted in mono-allelic gene editing and avoidance of undesired mutagenesis. In conclusion: DNA repair defects associated with FANCF deficiency do not prohibit CRISPR/Cas9 gene correction. Our data provide a solid basis for the application of pre-clinical models to further explore the potential of gene editing against FA, with the eventual aim to obtain therapeutic strategies against bone marrow failure.
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Registered trials
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