ArticleHematology. American Society of Hematology. Education Program2023
Gene therapy for sickle cell disease.
Article in Hematology. American Society of Hematology. Education Program, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.
What it found
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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
8 citing papers in PubMed.
- Splenectomy Impact and Outcome Among Patients With Sickle Cell Disease: A Cross-Sectional Study.Health science reports · 2026Article
- CRISPR-Cas9-mediated therapeutics: Current clinical trials and therapy approval landscape to treat human diseases.Molecular therapy. Nucleic acids · 2026Review
- Sickle Cell Disease: Treatment Options of the Past, Present, and Future.The American journal of nursing · 2026Review
- MeMAGEN: A Phase IIa/IIb open-label trial of memantine testing safety and tolerability in sickle cell patients.HemaSphere · 2026Article
- Review
- Clinical data comparison for FDA-approved gene therapies in sickle cell disease.Experimental biology and medicine (Maywood, N.J.) · 2025Article
- Current Strategies for Increasing Knock-In Efficiency in CRISPR/Cas9-Based Approaches.International journal of molecular sciences · 2024Review
- Cardiovascular Consequences of Sickle Cell Disease.Journal of blood medicine · 2024Article
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
No grant is acknowledged in the PubMed record.
Abstract
Sickle cell disease (SCD) is potentially curable after allogeneic hematopoietic stem cell transplantation (HSCT) or autologous HSCT after ex vivo genetic modification. Autologous HSCT with gene therapy has the potential to overcome many of the limitations of allogeneic HSCT that include the lack of suitable donors, graft-versus-host disease, the need for immune suppression, and the potential for graft rejection. Significant progress in gene therapy for SCD has been made over the past several decades, now with a growing number of clinical trials investigating various gene addition and gene editing strategies. Available results from a small number of patients, some with relatively short follow-up, are promising as a potentially curative strategy, with current efforts focused on continuing to improve the efficacy, durability, and safety of gene therapies for the cure of SCD.
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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.