ReviewCell reports. Medicine2025
Gene therapy for hemoglobinopathies: Clinical trial results and biology of hematopoietic stem cell and the bone marrow niche.
Review in Cell reports. Medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 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
2 citing papers in PubMed.
- Clinical Trial Landscape of Gene-Edited Autologous Hematopoietic Stem Cells for Hemoglobinopathies and Immunodeficiencies.International journal of molecular sciences · 2026Review
- Curative Approach to the Treatment of Beta-Thalassemia and Sickle Cell Disease with Hematopoietic Stem Cell Transplantation.Journal of clinical medicine · 2026Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Hemoglobinopathies, including beta-thalassemia (Bthal) and sickle cell disease (SCD), are among the most prevalent inherited blood disorders. Genetic mutations affecting hemoglobin synthesis result in severe anemia and multi-organ complications. The development of gene therapy (GT) aimed at correcting or modifying the hematopoietic system, although initially impaired by several limitations, has accomplished the marketing authorization of two hematopoietic stem cell (HSC) medicinal products, engineered by lentiviral vector gene addition and by CRISPR-Cas9 gene editing. Nonetheless, the success of these approaches stimulates a critical revision of our knowledge of HSC biology and bone marrow (BM) microenvironment in these diseases. Here, we review the clinical application of GT by gene addition and gene editing, and the novel findings about HSC and BM niche features and function in hemoglobinopathies. The identification of defective networks in HSC-niche is examined with the perspective of developing combined strategies to ameliorate the BM microenvironment to better support the genetically corrected cells.
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Identifiers
What Socratic 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 Socratic graph.