ArticleStem cells and development2016
CRISPR/Cas9 Targeted Gene Editing and Cellular Engineering in Fanconi Anemia.
Article in Stem cells and development, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
17 citing papers in PubMed, 28 citations in OpenAlex.
- CRISPR/Cas9-Mediated Correction of the SLC25A38 Mutation in a Patient With Sideroblastic Anemia.The journal of gene medicine · 2026Article
- [Advancements in CRISPR-Cas9 for Fanconi anemia].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2025Review
- Pediatric Bone Marrow Failure: A Broad Landscape in Need of Personalized Management.Journal of clinical medicine · 2023Review
- Adenine base editing efficiently restores the function of Fanconi anemia hematopoietic stem and progenitor cells.Nature communications · 2022Article
- Review
- CRISPR-Cas9: A Preclinical and Clinical Perspective for the Treatment of Human Diseases.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Review
- Nonsense Suppression Therapy: New Hypothesis for the Treatment of Inherited Bone Marrow Failure Syndromes.International journal of molecular sciences · 2020Review
- CRISPR to fix bad blood: a new tool in basic and clinical hematology.Haematologica · 2019Review
- [Establishment of a stable HEK293T cell line with c.392G>T (p.131G>V) mutation site knockout inNan fang yi ke da xue xue bao = Journal of Southern Medical University · 2019Article
- Effective CRISPR/Cas9-mediated correction of a Fanconi anemia defect by error-prone end joining or templated repair.Scientific reports · 2019Article
- Time-Restricted PiggyBac DNA Transposition by Transposase Protein Delivery Using Lentivirus-Derived Nanoparticles.Molecular therapy. Nucleic acids · 2018Article
- Integrative omics for health and disease.Nature reviews. Genetics · 2018Review
- Emerging Therapeutic Approaches for Diamond Blackfan Anemia.Current gene therapy · 2018Review
- CRISPR Editing in Biological and Biomedical Investigation.Journal of cellular biochemistry · 2017Review
- CRISPR/Cas9-Mediated Correction of the FANCD1 Gene in Primary Patient Cells.International journal of molecular sciences · 2017Article
- Significance of functional disease-causal/susceptible variants identified by whole-genome analyses for the understanding of human diseases.Proceedings of the Japan Academy. Series B, Physical and biological sciences · 2017Review
- CRISPR/Cas9-based genetic correction for recessive dystrophic epidermolysis bullosa.NPJ Regenerative medicine · 2016Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
11 authors at 1 institution in 1 country.
Funding
Abstract
The ability to rationally target disease-causing mutations has been made possible with programmable nucleases with the CRISPR/Cas9 system representing a facile platform for individualized gene-based medicine. In this study we employed footprint free reprogramming of fibroblasts from a patient with mutations to the Fanconi anemia I (FANCI) gene to generate induced pluripotent stem cells (iPSC). This process was accomplished without gene complementation and the resultant iPSC were able to be gene corrected in a robust manner using the Cas9 nickase. The self-renewing iPSC that were maintained under feeder free conditions were differentiated into cells with characteristics of definitive hematopoiesis. This defined and highly efficient procedure employed small molecule modulation of the hematopoietic differentiation pathway and a vascular induction technique to generate hematopoietic progenitors. In sum, our results demonstrate the ability to induce patient derived FA cells to pluripotency for patient specific therapeutic cell derivation.
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.