ArticleEMBO molecular medicine2017
Therapeutic gene editing in CD34
Article in EMBO molecular medicine, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 31 papers.
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The trial behind it
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Who cites it
31 citing papers in PubMed, 59 citations in OpenAlex.
- Removal of TREX1 activity enhances CRISPR-Cas9-mediated homologous recombination.Nature biotechnology · 2025Article
- Role of the mesenchymal stromal cells in bone marrow failure of Fanconi Anemia patients.Frontiers in cell and developmental biology · 2024Article
- Research progress on the fanconi anemia signaling pathway in non-obstructive azoospermia.Frontiers in endocrinology · 2024Review
- Illustrative Potency Assay Examples from Approved Therapies.Advances in experimental medicine and biology · 2023Article
- Adenine base editing efficiently restores the function of Fanconi anemia hematopoietic stem and progenitor cells.Nature communications · 2022Article
- Synergistic prostaglandin E synthesis by myeloid and endothelial cells promotes fetal hematopoietic stem cell expansion in vertebrates.The EMBO journal · 2022Article
- Site-specific genome editing in treatment of inherited diseases: possibility, progress, and perspectives.Medical review (2021) · 2022Review
- Improved engraftment and therapeutic efficacy by human genome-edited hematopoietic stem cells with Busulfan-based myeloablation.Molecular therapy. Methods & clinical development · 2022Article
- CRISPR/Cas9-Directed Gene Trap Constitutes a Selection System for CorrectedInternational journal of molecular sciences · 2022Article
- Precise somatic genome editing for treatment of inborn errors of immunity.Frontiers in immunology · 2022Review
- Translational research for bone marrow failure patients.Experimental hematology · 2022Review
- Article
- Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency.Molecular therapy. Methods & clinical development · 2021Article
- Review
- Advances in genome editing: the technology of choice for precise and efficient β-thalassemia treatment.Gene therapy · 2021Review
- Corrigendum: Targeted Gene Delivery: Where to Land.Frontiers in genome editing · 2021Article
- Baboon Envelope Pseudotyped "Nanoblades" Carrying Cas9/gRNA Complexes Allow Efficient Genome Editing in Human T, B, and CD34Frontiers in genome editing · 2021Article
- CRISPR/Cas9 for the treatment of haematological diseases: a journey from bacteria to the bedside.British journal of haematology · 2021Review
- Targeted gene therapy into a safe harbor site in human hematopoietic progenitor cells.Gene therapy · 2020Article
- Beyond chimerism analysis: methods for tracking a new generation of cell-based medicines.Bone marrow transplantation · 2020Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
14 authors at 6 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Gene targeting constitutes a new step in the development of gene therapy for inherited diseases. Although previous studies have shown the feasibility of editing fibroblasts from Fanconi anemia (FA) patients, here we aimed at conducting therapeutic gene editing in clinically relevant cells, such as hematopoietic stem cells (HSCs). In our first experiments, we showed that zinc finger nuclease (ZFN)-mediated insertion of a non-therapeutic EGFP-reporter donor in the
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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.