ReviewStem cells and development2017
Gene Therapy with Hematopoietic Stem Cells: The Diseased Bone Marrow's Point of View.
Review in Stem cells and development, 2017. 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
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.
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.
- Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study.The Lancet. Haematology · 2019Trial
- Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion.Haematologica · 2018Trial
- Safety, efficacy and feasibility of GCSF and plerixafor based CD34 mobilization for backup autologous stem cell collection in patients undergoing alternate donor hematopoietic stem cell transplant for sickle cell disease: Take away lessons for gene therapy.Bone marrow transplantation · 2026Article
- Gene therapy for hemoglobinopathies: Clinical trial results and biology of hematopoietic stem cell and the bone marrow niche.Cell reports. Medicine · 2025Review
- Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency.Nature medicine · 2024Article
- Gene modification therapies for hereditary diseases in the fetus.Prenatal diagnosis · 2023Review
- Targeting the Hematopoietic Stem Cell Niche in β-Thalassemia and Sickle Cell Disease.Pharmaceuticals (Basel, Switzerland) · 2022Review
- Clinical genome editing to treat sickle cell disease-A brief update.Frontiers in medicine · 2022Review
- Disease severity impacts plerixafor-mobilized stem cell collection in patients with sickle cell disease.Blood advances · 2021Article
- Article
- Immune Reconstitution After Gene Therapy Approaches in Patients With X-Linked Severe Combined Immunodeficiency Disease.Frontiers in immunology · 2020Review
- Bone Marrow-Derived Mesenchymal Stromal Cells: A Novel Target to Optimize Hematopoietic Stem Cell Transplantation Protocols in Hematological Malignancies and Rare Genetic Disorders.Journal of clinical medicine · 2019Review
- Low-Dose Busulfan Reduces Human CD34Molecular therapy. Methods & clinical development · 2019Article
- Bone marrow stromal cells from β-thalassemia patients have impaired hematopoietic supportive capacity.The Journal of clinical investigation · 2019Article
- A comparison of intrauterine hemopoietic cell transplantation and lentiviral gene transfer for the correction of severe β-thalassemia in a HbbTh3/+ murine model.Experimental hematology · 2018Article
- Molecular basis of β thalassemia and potential therapeutic targets.Blood cells, molecules & diseases · 2018Review
- Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors at 3 institutions in 1 country.
Funding
Abstract
When considering inherited diseases that can be treated by gene transfer into hematopoietic stem cells (HSCs), there are only two in which the HSC and progenitor cell distribution inside the bone marrow and its microenvironment are exactly the same as in a healthy subject: metachromatic leukodystrophy (MLD) and adrenoleukodystrophy (ALD). In all other settings [X-linked severe combined immunodeficiency (X-SCID), adenosine deaminase deficiency, Wiskott-Aldrich syndrome, and β-hemoglobinopathies], the bone marrow content of the different stem and precursor cells and the cells' relationship with the stroma have very specific characteristics. These peculiarities can influence the cells' harvesting and behavior in culture, and the postgraft uptake and further behavior of the gene-modified hematopoietic/precursor cells. In the present mini-review, we shall briefly summarize these characteristics and outline the possible consequences and challenges.
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.