ArticleJournal of translational medicine2015
Cysteamine treatment restores the in vitro ability to differentiate along the osteoblastic lineage of mesenchymal stromal cells isolated from bone marrow of a cystinotic patient.
Article in Journal of translational medicine, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.
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Who cites it
14 citing papers in PubMed, 23 citations in OpenAlex.
- Cystinosis-associated metabolic bone disease: pathogenesis and outcome.Pediatric nephrology (Berlin, Germany) · 2026Review
- The assessment and treatment of the musculoskeletal manifestations of cystinosis.Frontiers in nephrology · 2025Review
- Cortical impairment and reduced muscle mass in children and young adults with nephropathic cystinosis.Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research · 2024Article
- Alterations in bone metabolites with age in C57BL/6 mice model.Biogerontology · 2022Article
- Review
- Peripheral Blood Mononuclear Cells (PBMCs) to Dissect the Underlying Mechanisms of Bone Disease in Chronic Kidney Disease and Rare Renal Diseases.Current osteoporosis reports · 2021Review
- Drug Repurposing in Rare Diseases: An Integrative Study of Drug Screening and Transcriptomic Analysis in Nephropathic Cystinosis.International journal of molecular sciences · 2021Article
- Article
- Bone Disease in Nephropathic Cystinosis: Beyond Renal Osteodystrophy.International journal of molecular sciences · 2020Review
- Mitochondrial Dynamics of Proximal Tubular Epithelial Cells in Nephropathic Cystinosis.International journal of molecular sciences · 2019Article
- Management of bone disease in cystinosis: Statement from an international conference.Journal of inherited metabolic disease · 2019Article
- Effects of long-term cysteamine treatment in patients with cystinosis.Pediatric nephrology (Berlin, Germany) · 2019Review
- Skeletal Consequences of Nephropathic Cystinosis.Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research · 2018Article
- Skeletal implications and management of cystinosis: three case reports and literature review.BoneKEy reports · 2016Review
Corrections and comments
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Authors and funding
10 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundCystinosis is a rare autosomal recessive disease caused by mutations of the CTNS gene, which encodes for a lysosomal cystine/H(+) symporter. In mice, inactivation of the CTNS gene causes intralysosomal cystine accumulation and progressive organ damage that can be reversed, at least in part, by infusion of mesenchymal stromal cells (MSCs). Little is known on the mesenchymal compartment of cystinotic patients. The aim of the study was to test the phenotypical and functional properties of cystinotic MSCs (Cys-MSCs) isolated from bone marrow (BM) aspirate of a patient with nephropathic cystinosis.
methodsMorphology, proliferative capacity (measured as population doublings), immunophenotype (by flow-cytometry) and immunomodulatory properties (as phytohemagglutinin-induced peripheral blood mononuclear cell proliferation) were analyzed. The osteogenic differentiation potential of Cys-MSCs was evaluated by histological staining (alkaline phosphatase activity, Alzarin Red and von Kossa staining) spectrophotometry and Quantitative Reverse Transcriptase Polymerase Chain Reaction for osteigenic markers in the presence and in the absence of cysteamine. Cys-MSCs were compared with those isolated and expanded ex vivo from three healthy donors (HD-MSCs).
resultsDespite a slightly lower proliferative capacity, Cys-MSCs displayed a characteristic spindle-shaped morphology and similar immunephenotype as HD-MSCs. Cys-MSCs and HD-MSCs prevented proliferation of PHA-stimulated allogeneic peripheral blood mononuclear cells to the same extent. After in vitro induction into osteoblasts, Cys-MSCs showed reduced alkaline phosphatase (ALP) activity, calcium depositions and expression of ALP and collagen type 1. When Cys-MSCs were treated in vitro with increasing doses of cysteamine (50-100-200 μM/L) during the differentiation assay, recovery of Cys-MSCs differentiation capacity into osteoblasts was observed. No difference in adipogenic differentiation was found between Cys-MSCs and HD-MSCs.
conclusionsOur results indicate that, as compared to HD-MSCs, Cys-MSCs show reduced ability to differentiate into osteoblasts, which can be reverted after cysteamine treatment.
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