ReviewAnnual review of medicine2023
Diverse Approaches to Gene Therapy of Sickle Cell Disease.
Review in Annual review of medicine, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 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
13 citing papers in PubMed, 30 citations in OpenAlex.
- Challenges and limitations of mobilization and stem cell collection for gene therapy of sickle cell disease.Blood advances · 2025Review
- Emerging Therapies for Sickle Cell Disease: From Symptom Management to Curative Gene Therapy.Cureus · 2025Review
- Advancements in CRISPR/Cas systems for disease treatment.Acta pharmaceutica Sinica. B · 2025Review
- The role of nucleation in sickle cell pathophysiology: opportunities for innovative treatments.Annals of medicine and surgery (2012) · 2025Review
- Article
- Direct delivery of Cas-embedded cytosine base editors as ribonucleoprotein complexes for efficient and accurate editing of clinically relevant targets.Nucleic acids research · 2025Article
- CRISPR technology in human diseases.MedComm · 2024Review
- Rare genetic disorders in India: Current status, challenges, and CRISPR-based therapy.Journal of biosciences · 2024Review
- Enhancing regenerative medicine: the crucial role of stem cell therapy.Frontiers in neuroscience · 2024Review
- An α-chain modification rivals the effect of fetal hemoglobin in retarding the rate of sickle cell fiber formation.Scientific reports · 2023Article
- Successes and challenges in clinical gene therapy.Gene therapy · 2023Article
- Current challenges and new approaches to implementing optimal management of sickle cell disease in sub-Saharan Africa.Seminars in hematology · 2023Article
- Hematopoietic Stem Cell Transplantation in Sickle Cell Disease: A Multidimentional Review.Cell transplantationReview
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 at 2 institutions in 1 country.
Funding
Abstract
Sickle cell disease (SCD) results from a single base pair change in the sixth codon of the β-globin chain of hemoglobin, which promotes aggregation of deoxyhemoglobin, increasing rigidity of red blood cells and causing vaso-occlusive and hemolytic complications. Allogeneic transplant of hematopoietic stem cells (HSCs) can eliminate SCD manifestations but is limited by absence of well-matched donors and immune complications. Gene therapy with transplantation of autologous HSCs that are gene-modified may provide similar benefits without the immune complications. Much progress has been made, and patients are realizing significant clinical improvements in multiple trials using different approaches with lentiviral vector-mediated gene addition to inhibit hemoglobin aggregation. Gene editing approaches are under development to provide additional therapeutic opportunities. Gene therapy for SCD has advanced from an attractive concept to clinical reality.
Indexed as
Identifiers
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.