ArticleNature communications2024
Development of pathophysiologically relevant models of sickle cell disease and β-thalassemia for therapeutic studies.
Article in Nature communications, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers, 1 of them a synthesis that pooled it.
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Who cites it
6 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Towards the standardization of human platelet lysate production and its comparison to fetal bovine serum for human hematopoietic cell culture: a scoping review.Frontiers in toxicology · 2025Pooled it
- Modeling Genetic Diversity in Sickle Cell Disease Reveals Heterogeneous Responses to HbF-Inducing Therapies.bioRxiv : the preprint server for biology · 2026Article
- Roxadustat induced fetal hemoglobin in anemic CKD patients: a dual-center real-world observational cohort study.BMC nephrology · 2026Observational
- CRISPR editing of HPFH3 genotype induces γ-globin expression and reverses sickle cell disease and β-thalassemia phenotypes.Stem cell research & therapy · 2025Article
- CRISPR/Cas9-engineering of Kell null erythrocytes to unveil host targeted irresistible antimalarial.Communications biology · 2025Article
- Sickle cell disease in India: the journey and hope for the future.Hematology. American Society of Hematology. Education Program · 2024Review
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Authors and funding
18 authors.
Funding
Abstract
Ex vivo cellular system that accurately replicates sickle cell disease and β-thalassemia characteristics is a highly sought-after goal in the field of erythroid biology. In this study, we present the generation of erythroid progenitor lines with sickle cell disease and β-thalassemia mutation using CRISPR/Cas9. The disease cellular models exhibit similar differentiation profiles, globin expression and proteome dynamics as patient-derived hematopoietic stem/progenitor cells. Additionally, these cellular models recapitulate pathological conditions associated with both the diseases. Hydroxyurea and pomalidomide treatment enhanced fetal hemoglobin levels. Notably, we introduce a therapeutic strategy for the above diseases by recapitulating the HPFH3 genotype, which reactivates fetal hemoglobin levels and rescues the disease phenotypes, thus making these lines a valuable platform for studying and developing new therapeutic strategies. Altogether, we demonstrate our disease cellular systems are physiologically relevant and could prove to be indispensable tools for disease modeling, drug screenings and cell and gene therapy-based applications.
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