ReviewCytotherapy2018
Gene therapy for sickle cell disease: An update.
Review in Cytotherapy, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 51 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
51 citing papers in PubMed.
- Sickle Cell Disease: Treatment Options of the Past, Present, and Future.The American journal of nursing · 2026Review
- In Silico Post-screening of Anti-polymerization Agents to Treat Sickle Cell Disease.bioRxiv : the preprint server for biology · 2025Article
- Sickle Cell Disease and Gene Therapy Among African Americans: A Dilemma and Challenge.Journal of racial and ethnic health disparities · 2025Review
- The dawn of a cure for sickle cell disease through CRISPR-based treatment: A critical test of equity in public health genomics.Annals of human genetics · 2025Review
- DRBP-EDP: classification of DNA-binding proteins and RNA-binding proteins using ESM-2 and dual-path neural network.NAR genomics and bioinformatics · 2025Article
- Genome editing strategies for targeted correction of β-globin mutation in sickle cell disease: From bench to bedside.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- CRISPR/Cas System: A Powerful Strategy to Improve Monogenic Human Diseases as Therapeutic Delivery; Current Applications and Challenges.Current gene therapy · 2025Review
- Sickle cell disease: understanding pathophysiology, clinical features and advances in gene therapy approaches.Frontiers in pharmacology · 2025Review
- UM171 enhances fitness and engraftment of gene-modified hematopoietic stem cells from patients with sickle cell disease.Blood advances · 2024Article
- Advancing life: innovative approaches to enhance survival in sickle cell anemia patients.Annals of medicine and surgery (2012) · 2024Review
- Pulmonary Hypertension in Sickle Cell Disease: Novel Findings of Gene Polymorphisms Related to Pathophysiology.International journal of molecular sciences · 2024Review
- Biophysical chemistry behind sickle cell anemia and the mechanism of voxelotor action.Scientific reports · 2024Article
- Current challenges and new approaches to implementing optimal management of sickle cell disease in sub-Saharan Africa.Seminars in hematology · 2023Article
- Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI).Gene therapy · 2023Review
- Sublingual Microcirculation Specificity of Sickle Cell Patients: Morphology of the Microvascular Bed, Blood Rheology, and Local Hemodynamics.International journal of molecular sciences · 2023Article
- Genotypic and Phenotypic Composition of Sickle Cell Disease in the Arab Population - A Systematic Review.Pharmacogenomics and personalized medicine · 2023Review
- Pseudotyped Viruses for Retroviruses.Advances in experimental medicine and biology · 2023Article
- Article
- Surveillance for Sickle Cell Disease - Sickle Cell Data Collection Program, Two States, 2004-2018.Morbidity and mortality weekly report. Surveillance summaries (Washington, D.C. : 2002) · 2022Article
- A landscape analysis and discussion of value of gene therapies for sickle cell disease.Expert review of pharmacoeconomics & outcomes research · 2022Article
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.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Sickle cell disease (SCD) is one of the most common life-threatening monogenic diseases affecting millions of people worldwide. Allogenic hematopietic stem cell transplantation is the only known cure for the disease with high success rates, but the limited availability of matched sibling donors and the high risk of transplantation-related side effects force the scientific community to envision additional therapies. Ex vivo gene therapy through globin gene addition has been investigated extensively and is currently being tested in clinical trials that have begun reporting encouraging data. Recent improvements in our understanding of the molecular pathways controlling mammalian erythropoiesis and globin switching offer new and exciting therapeutic options. Rapid and substantial advances in genome engineering tools, particularly CRISPR/Cas9, have raised the possibility of genetic correction in induced pluripotent stem cells as well as patient-derived hematopoietic stem and progenitor cells. However, these techniques are still in their infancy, and safety/efficacy issues remain that must be addressed before translating these promising techniques into clinical practice.
Indexed as
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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.