ReviewCytotherapy2025
Gene therapy for sickle cell disease: recent advances, clinical trials and future directions.
Review in Cytotherapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 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
12 citing papers in PubMed.
- Sickle Cell Disease: From Ancient Origins to Modern Breakthroughs in Gene Therapy.Biomedicines · 2026Review
- Gene Therapy for β-Haemoglobinopathies: From Molecular Correction to Curative Medicine.Biomedicines · 2026Review
- Beyond hematopoietic stem cell transplantation: positioning Africa for scalable uptake of innovative sickle cell therapies.BMJ global health · 2026Article
- Which religious and personal characteristics predict attitudes toward gene editing? Findings from a survey of 4,939 adults in the U.S.Journal of community genetics · 2026Article
- Coping Skills, Hospitalizations, and Hopefulness in Youths with Sickle Cell Disease Treated in a Regional Outpatient Comprehensive Pediatric Center.Children (Basel, Switzerland) · 2026Article
- Genetics and Genomics in Sickle Cell Disease in Africa.American journal of hematology · 2026Review
- Unravelling the Immunological Enigma of Sickle Cell Disease: Current Understanding and Future Directions.Immunology · 2026Review
- miRNA-Mediated Regulation of γ-Globin to β-Globin Switching: Therapeutic Potential in β-Hemoglobinopathies.International journal of molecular sciences · 2026Review
- Blurring boundaries or structural coupling? Revisiting the science-medicine interface through the analogy of politics and law.Frontiers in sociology · 2026Article
- Gene Therapy of Beta Hemoglobinopathies.Biomedicines · 2025Review
- Knowledge mapping and bibliometric insights into gene therapy for rare inherited hematologic pathologies: focus on sickle cell disease, hemophilia, and thalassemia.Orphanet journal of rare diseases · 2025Article
- Emerging Therapies for Sickle Cell Disease: From Symptom Management to Curative Gene Therapy.Cureus · 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
Sickle cell disease (SCD) is the most common inherited blood disorder worldwide, impacting millions and imposing severe healthcare challenges, particularly in resource-limited regions. Current treatments have variable efficacy and require lifelong adherence. Allogeneic Hematopoietic Stem Cell Transplantation can be curative but comes with significant side effects and limited donor availability limits its widespread applicability. Gene therapy, by addressing the root genetic causes, offers a revolutionary alternative. This article discusses the molecular mechanisms of SCD and β-thalassemia and highlights advancements in gene therapy, such as gene addition via lentiviral vectors and gene editing with CRISPR/Cas9 technology. Clinical trials have brought about significant progress but challenges remain, including leukemogenesis, delivery efficiency and cost. Future efforts must focus on enhancing efficiency, reducing costs, developing nongenotoxic conditioning regimens and methods for in vivo application.
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.