ArticleBlood2020
Hematopoietic stem cell function in β-thalassemia is impaired and is rescued by targeting the bone marrow niche.
Article in Blood, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers.
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Who cites it
27 citing papers in PubMed.
- Update of the decision-making algorithm on selecting transfusion-dependent β-thalassemic patients for gene therapy approaches: joint consensus report on behalf of EHA-specialized working group and EBMT hemoglobinopathies working party.Bone marrow transplantation · 2026Article
- Article
- Rapidly dissolving biomaterials for high-efficiency viral transduction.Acta biomaterialia · 2026Article
- Greening Bone Healing: The Emerging Role of Plant-Derived Exosome-Like Nanoparticles in Osteoporosis and Osteoarthritis Therapy.International journal of nanomedicine · 2026Review
- Biochemical Predictors and Clinical Characteristics for the Development of Cytopenia and Bone Marrow Involvement Among Patients With Primary Hyperparathyroidism.International journal of endocrinology · 2026Article
- Gene therapy for hemoglobinopathies: Clinical trial results and biology of hematopoietic stem cell and the bone marrow niche.Cell reports. Medicine · 2025Review
- Imbalanced TGFβ signalling and autophagy drive erythroid priming of hematopoietic stem cells in β-thalassemia.Nature communications · 2025Article
- Single-cell RNA sequencing identifies the expression of hemoglobin in chondrocyte cell subpopulations in osteoarthritis.BMC molecular and cell biology · 2024Article
- Article
- Exploring the bone marrow micro environment in thalassemia patients: potential therapeutic alternatives.Frontiers in immunology · 2024Review
- The impact of gut microbial signals on hematopoietic stem cells and the bone marrow microenvironment.Frontiers in immunology · 2024Review
- Diversity in the bone marrow niche: Classic and novel strategies to uncover niche composition.British journal of haematology · 2022Review
- Targeting the Hematopoietic Stem Cell Niche in β-Thalassemia and Sickle Cell Disease.Pharmaceuticals (Basel, Switzerland) · 2022Review
- The EHA Research Roadmap: Hematopoietic Stem Cell Gene Therapy.HemaSphere · 2022Article
- Relationship between indices of circulating blood cells and bone homeostasis in osteoporosis.Frontiers in endocrinology · 2022Article
- Article
- Editing outside the body: Ex vivo gene-modification for β-hemoglobinopathy cellular therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Review
- Ineffective Erythropoiesis in β-Thalassaemia: Key Steps and Therapeutic Options by Drugs.International journal of molecular sciences · 2021Review
- Review
- Gene therapy using haematopoietic stem and progenitor cells.Nature reviews. Genetics · 2021Review
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Authors and funding
12 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Hematopoietic stem cells (HSCs) are regulated by signals from the bone marrow (BM) niche that tune hematopoiesis at steady state and in hematologic disorders. To understand HSC-niche interactions in altered nonmalignant homeostasis, we selected β-thalassemia, a hemoglobin disorder, as a paradigm. In this severe congenital anemia, alterations secondary to the primary hemoglobin defect have a potential impact on HSC-niche cross talk. We report that HSCs in thalassemic mice (th3) have an impaired function, caused by the interaction with an altered BM niche. The HSC self-renewal defect is rescued after cell transplantation into a normal microenvironment, thus proving the active role of the BM stroma. Consistent with the common finding of osteoporosis in patients, we found reduced bone deposition with decreased levels of parathyroid hormone (PTH), which is a key regulator of bone metabolism but also of HSC activity. In vivo activation of PTH signaling through the reestablished Jagged1 and osteopontin levels correlated with the rescue of the functional pool of th3 HSCs by correcting HSC-niche cross talk. Reduced HSC quiescence was confirmed in thalassemic patients, along with altered features of the BM stromal niche. Our findings reveal a defect in HSCs in β-thalassemia induced by an altered BM microenvironment and provide novel and relevant insight for improving transplantation and gene therapy approaches.
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