ReviewBlood advances2025
To pursue gene therapy or not? Is it feasible after graft failure in allogeneic hematopoietic cell transplant recipients.
Review in Blood advances, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers, 1 of them a synthesis that pooled it.
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
4 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Gene Therapy for Sickle Cell Disease: Practice Recommendations from the American Society for Transplantation and Cellular Therapy and the International Society for Cell & Gene Therapy.Transplantation and cellular therapy · 2026Guideline
- Alternative conditioning regimens for hemoglobinopathy gene therapy: balancing efficacy, toxicity, and the next frontier.Blood advances · 2026Review
- Curative Approach to the Treatment of Beta-Thalassemia and Sickle Cell Disease with Hematopoietic Stem Cell Transplantation.Journal of clinical medicine · 2026Review
- Gene Therapy of Beta Hemoglobinopathies.Biomedicines · 2025Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors.
Funding
Abstract
abstractTwo autologous hematopoietic stem cell (HSC)-based gene therapies (GTs) are now commercially available for severe sickle cell disease and transfusion-dependent β-thalassemia. However, the safety and efficacy of a subsequent autologous HSC-based GT after graft failure with a previous allogeneic hematopoietic cell transplant (HCT) remains unclear. Some individuals who have experienced a failed first attempt at a potentially curative therapy might seek a second opportunity for cure via GT. In this article, we discuss various factors related to patient and HSC health that may influence feasibility, and shared decision-making regarding whether an individual who has previously received an allogeneic HCT and experienced graft failure could consider an autologous GT. Exposure to chronic inflammatory stress and conditioning chemotherapy may compromise HSC fitness, reduce hematopoietic reserve, accelerate HSC aging, and promote the accumulation of deleterious genetic mutations, all of which may adversely affect the safety and efficacy of the GT.
Indexed as
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.