ReviewFrontiers in genome editing2021
Gene Editing of Hematopoietic Stem Cells: Hopes and Hurdles Toward Clinical Translation.
Review in Frontiers in genome editing, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06959771 (Base Editing Hematopoietic Stem Cell and BE T Cell Gene Therapy for CD40L-HyperIgM Syndrome-Single Patient Study), which is not on this map. Cited by 32 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
Base Editing Hematopoietic Stem Cell and BE T Cell Gene Therapy for CD40L-HyperIgM Syndrome-Single Patient Study
Who cites it
32 citing papers in PubMed, 41 citations in OpenAlex.
- mRNA Lipid Nanoparticles for Cell Engineering in Vivo and in Vitro: Current Applications and Future Directions.MedComm · 2026Review
- Immunomodulatory Agents and Factors in Poor Hematopoietic Stem Cell Mobilization: Current Strategies and Future Prospects.Stem cell reviews and reports · 2026Review
- CRISPR/Cas9 gene editing in hematopoietic stem and progenitor cells to accelerate the translation of cellular therapies and immunotherapies.Journal of translational research · 2026Article
- Clinical translation of CRISPR-Cas9 therapeutics in cancer and inherited genetic disorders.Frontiers in genome editing · 2026Review
- Base and Prime Editing for Inherited Retinal Diseases: Delivery Platforms, Safety, Efficacy, and Translational Perspectives.Pharmaceutics · 2025Review
- Gene-edited hematopoietic stem cells for leukemia and lymphoma treatment: a systematic review of preclinical and translational evidence.Discover oncology · 2025Review
- Gene editing in hematopoietic stem cells by co-delivery of Cas9/sgRNA ribonucleoprotein and templates for homology-directed repair in 'all-in-one' lentivirus-derived nanoparticles.Nucleic acids research · 2025Article
- Clinical hematopoietic stem cell-based gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Advanced delivery systems for gene editing: A comprehensive review from the GenE-HumDi COST Action Working Group.Molecular therapy. Nucleic acids · 2025Review
- Review
- Gene Therapy Techniques and Delivery Methods (Review).Sovremennye tekhnologii v meditsine · 2025Review
- Integrating Mitochondrial Biology into Innovative Cell Therapies for Neurodegenerative Diseases.Brain sciences · 2024Review
- CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Functional screening in human HSPCs identifies optimized protein-based enhancers of Homology Directed Repair.Nature communications · 2024Article
- Review
- Mobilization-based engraftment of haematopoietic stem cells: a new perspective for chemotherapy-free gene therapy and transplantation.British medical bulletin · 2023Review
- Lipid nanoparticles allow efficient and harmless ex vivo gene editing of human hematopoietic cells.Blood · 2023Article
- Bioinformatic and literature assessment of toxicity and allergenicity of a CRISPR-Cas9 engineered gene drive to control Anopheles gambiae the mosquito vector of human malaria.Malaria journal · 2023Article
- An improved medium formulation for efficientMolecular therapy. Methods & clinical development · 2023Article
- Gene editing innovations and their applications in cardiomyopathy research.Disease models & mechanisms · 2023Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors at 5 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
In the field of hematology, gene therapies based on integrating vectors have reached outstanding results for a number of human diseases. With the advent of novel programmable nucleases, such as CRISPR/Cas9, it has been possible to expand the applications of gene therapy beyond semi-random gene addition to site-specific modification of the genome, holding the promise for safer genetic manipulation. Here we review the state of the art of
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.