ReviewFrontiers in pharmacology2025
Sickle cell disease: understanding pathophysiology, clinical features and advances in gene therapy approaches.
Review in Frontiers in pharmacology, 2025. 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
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
8 citing papers in PubMed.
- Cerebral fat embolism syndrome in sickle cell disease: a case highlighting the predominance of susceptibility-weighted imaging findings and literature review.Radiology case reports · 2026Article
- Vitamin D Deficiency as a Potential Modifier of Hematological Outcomes and Vaso-Occlusive Crisis Frequency in Sickle Cell Disease: Evidence from a Sudanese Cohort.Biomedicines · 2026Article
- Efficacy of curcumin and resveratrol in the synergistic activation of foetal haemoglobin in sickle cell disease.The Indian journal of medical research · 2026Article
- Global Prevalence of Alloimmunization in Adults with Sickle Cell Disease Receiving Red Blood Cell Transfusions: A Systematic Review and Meta-Analysis.Journal of clinical medicine · 2026Review
- More than malaria: ExploringCurrent research in parasitology & vector-borne diseases · 2026Review
- Temporal trends and determinants of ED visits in sickle cell disease: a multicenter EHR study from Saudi Arabia (2016-2021).Frontiers in medicine · 2026Article
- The Molecular Pathology of Non-Malignant Haematological Disease.British journal of biomedical science · 2026Review
- Clinical pathway implementation for adult sickle cell vaso-occlusive crises: Impact on healthcare utilization, opioid prescribing, and costs.Annals of Saudi medicineArticle
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
11 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Sickle cell disease (SCD) is an inherited blood disorder marked by the production of abnormal hemoglobin, leading to the distortion-or sickling-of red blood cells. The SCD arises from a single-point mutation that substitutes glutamic acid with valine at the sixth codon of the β-globin chain in hemoglobin. This substitution promotes deoxyhemoglobin aggregation, elevating red blood cell stiffness, and triggering vaso-occlusive and hemolytic repercussions. To explore therapeutic advances in tackling this disease, this review analyzed articles published from January 2015 to January 2025 using the three databases using relevant keywords focusing on SCD and advancement in therapy. It was found that allogeneic hematopoietic stem cell (HSC) transplantation can alleviate symptoms but is limited by a shortage of well-matched donors and immunological challenges. In contrast, autologous gene-modified HSC transplantation via gene therapy offers comparable therapeutic benefits without associated immunological complications. Clinical trials utilizing lentiviral vector-mediated gene insertion have demonstrated promising therapeutic outcomes by preventing hemoglobin aggregation. Emerging gene editing approaches such as CRISPR/Cas9 are expanding treatment options, marking the transition of SCD gene therapy from theoretical concept to clinical application.
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.