ArticleHuman gene therapy. Clinical development2017
Bone Marrow as a Hematopoietic Stem Cell Source for Gene Therapy in Sickle Cell Disease: Evidence from Rhesus and SCD Patients.
Article in Human gene therapy. Clinical development, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 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
20 citing papers in PubMed, 33 citations in OpenAlex.
- Clinical outcomes of lentiviral vector gene therapy for sickle cell disease.Blood advances · 2026Trial
- Novel lineage depletion preserves autologous blood stem cells for gene therapy of Fanconi anemia complementation group A.Haematologica · 2018Trial
- Challenges and limitations of mobilization and stem cell collection for gene therapy of sickle cell disease.Blood advances · 2025Review
- Gene therapy for hemoglobinopathies: Clinical trial results and biology of hematopoietic stem cell and the bone marrow niche.Cell reports. Medicine · 2025Review
- Immune modulation permits tolerance and engraftment in a murine model of late-gestation transplantation.Blood advances · 2024Article
- Small volume bone marrow aspirates with high progenitor cell concentrations maximize cell therapy dose manufacture and substantially reduce donor hemoglobin loss.BMC medicine · 2023Article
- Review
- Disease severity impacts plerixafor-mobilized stem cell collection in patients with sickle cell disease.Blood advances · 2021Article
- Preclinical evaluation for engraftment of CD34Cell reports. Medicine · 2021Article
- Genetic therapies for the first molecular disease.The Journal of clinical investigation · 2021Review
- Gene Therapy of the Hemoglobinopathies.HemaSphere · 2020Review
- Article
- Review
- Bone marrow characterization in sickle cell disease: inflammation and stress erythropoiesis lead to suboptimal CD34 recovery.British journal of haematology · 2019Article
- Curing Hemoglobinopathies: Challenges and Advances of Conventional and New Gene Therapy Approaches.Mediterranean journal of hematology and infectious diseases · 2019Review
- Stem cell transplantation in sickle cell disease: therapeutic potential and challenges faced.Expert review of hematology · 2018Review
- Serum-free Erythroid Differentiation for Efficient Genetic Modification and High-Level Adult Hemoglobin Production.Molecular therapy. Methods & clinical development · 2018Article
- Safety and efficacy of plerixafor dose escalation for the mobilization of CD34Haematologica · 2018Article
- Hematopoietic stem cell mobilization with plerixafor in sickle cell disease.Haematologica · 2018Article
- The effect of bone marrow mesenchymal stem cells on recovery of skeletal muscle after neurotization surgery in rat.Iranian journal of basic medical sciences · 2018Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
8 authors at 1 institution in 1 country.
Funding
Abstract
Steady state bone marrow (BM) is the preferred hematopoietic stem cell (HSC) source for gene therapy in sickle cell disease (SCD) due to the recognized risk of vaso-occlusive crisis during granulocyte colony-stimulating factor mobilization. We previously established clinically relevant HSC gene transfer in the rhesus model following transplantation of mobilized peripheral blood (PB) CD34
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.