ReviewBlood advances2026
Alternative conditioning regimens for hemoglobinopathy gene therapy: balancing efficacy, toxicity, and the next frontier.
Review in Blood advances, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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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
0 citing papers in PubMed.
No citing paper in PubMed yet.
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2 authors.
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Abstract
abstractGene therapy for sickle cell disease and transfusion-dependent thalassemia is now widely available, yet its broader application is constrained by the need for effective and tolerable conditioning. This review examines the central role of conditioning in enabling durable engraftment of gene-modified hematopoietic stem cells, emphasizing the narrow therapeutic window between insufficient niche clearance and excessive toxicity. Busulfan remains the standard conditioning agent, supported by consistent engraftment and clinical efficacy, but its use is limited by acute and long-term toxicities, including infertility and potential genotoxicity. Emerging strategies aim to mitigate these risks. Reduced-intensity melphalan shows early promise but raises concerns regarding the durability of engraftment, whereas treosulfan offers a potentially safer profile, although data in the autologous setting are lacking. Antibody-based, nongenotoxic approaches represent a transformative direction but remain investigational. Refining conditioning through comparative trials and long-term follow-up will be critical to fully realize the curative potential of gene therapy.
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