ArticleDisease models & mechanisms2023
The future of sickle cell disease therapeutics rests in genomics.
Article in Disease models & mechanisms, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 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
15 citing papers in PubMed, 19 citations in OpenAlex.
- Rare disease genomics and justice: overview of a workshop at the Fondation Brocher, 22-24 January 2025.Journal of community genetics · 2026Article
- The role of hydroxyurea in modulating miRNA expression in sickle cell disease: molecular mechanisms and therapeutic implications.Annals of hematology · 2026Review
- Genetics and Genomics in Sickle Cell Disease in Africa.American journal of hematology · 2026Review
- Systematic Review of Non-Coding Genomic Variants in Globin and Non-Globin Clusters and Their Impact on Phenotypic Severity in Thalassemia and Sickle Cell Disease.Journal of clinical medicine · 2026Review
- Nanohydrogels for Diabetic Wound Healing: Mechanisms, Applications, and Future Perspectives.International journal of nanomedicine · 2026Review
- FLT1 and other candidate fetal haemoglobin modifying loci in sickle cell disease in African ancestries.Nature communications · 2025Article
- Pulmonary Function after Nonmyeloablative Hematopoietic Cell Transplant for Sickle Cell Disease.Annals of the American Thoracic Society · 2024Article
- Looking ahead: ethical and social challenges of somatic gene therapy for sickle cell disease in Africa.Gene therapy · 2024Review
- Pharmacogenomics of Drugs Used in β-Thalassemia and Sickle-Cell Disease: From Basic Research to Clinical Applications.International journal of molecular sciences · 2024Review
- Development of pathophysiologically relevant models of sickle cell disease and β-thalassemia for therapeutic studies.Nature communications · 2024Article
- The transatlantic fight against sickle cell disease: an interview with Marie Ojiambo.Disease models & mechanisms · 2023Article
- Supporting the translation of multiscale research in rare disease.Disease models & mechanisms · 2023Article
- Current challenges and new approaches to implementing optimal management of sickle cell disease in sub-Saharan Africa.Seminars in hematology · 2023Article
- Editorial: Building capacity for sickle cell disease research and healthcare.Frontiers in genetics · 2023Article
- Towards an appropriate African framework for public engagement with human genome editing: a call to synergistic action.Wellcome open 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
1 author at 1 institution in 1 country.
Funding
Abstract
Sickle cell disease (SCD) is the most-common monogenic recessive disease in humans, annually affecting almost 300,000 newborns worldwide, 75% of whom live in Africa. Genomics research can accelerate the development of curative therapies for SCD in three ways. First, research should explore the missing heritability of foetal haemoglobin (HbF) - the strongest known modifier of SCD clinical expression - among highly genetically heterogenous and understudied African populations, to provide novel therapeutics targets for HbF induction. Second, SCD research should invest in RNA therapies, either by using microRNA to target the production of HbF proteins by binding to the transcription machinery in a cell, or by directly mediating production of HbF or adult haemoglobin through injection of messenger RNA. Third, investigators should aim to identify currently unknown genetic risk factors for SCD cardiovascular complications, which will address mortality, particularly in adults. Now is the time for global research programs to uncover genomic keys to unlock SCD therapeutics.
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.