ArticleNature2014
Statin treatment rescues FGFR3 skeletal dysplasia phenotypes.
Article in Nature, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 114 papers, 1 of them a synthesis that pooled it.
What it found
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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.
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Who cites it
114 citing papers in PubMed, 1 synthesis or guideline pooled it, 207 citations in OpenAlex.
- Dyslipidemia Might Be Associated with an Increased Risk of Osteoarthritis.BioMed research international · 2020Pooled it
- A pilot study of gene testing of genetic bone dysplasia using targeted next-generation sequencing.Journal of human genetics · 2015Trial
- Achondroplasia: Current Disease-Modifying Therapies and Genome Editing Strategies.International journal of molecular sciences · 2026Review
- Induced pluripotent stem cells from discovery to translation.Nature medicine · 2026Review
- RAS/MAPK pathway modulation with simvastatin in children with Noonan syndrome: a multicentre, randomised, double-blind, placebo-controlled phase 3 trial.EClinicalMedicine · 2026Article
- Investigation of cartilage modification for controlling immune reaction against xenogeneic cartilage.Regenerative therapy · 2026Article
- Bridging skeletal stem cell diversity and human skeletal modeling.Journal of bone and mineral metabolism · 2026Review
- Advances in the mechanism and therapies of achondroplasia.Genes & diseases · 2025Review
- p75 neurotrophin receptor regulates craniofacial growth and morphology in postnatal development.Frontiers in cell and developmental biology · 2025Article
- In Vitro Evaluation of Exon Skipping in Disease-Specific iPSC-Derived Myocytes.Methods in molecular biology (Clifton, N.J.) · 2025Article
- Modeling of skeletal development and diseases using human pluripotent stem cells.Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research · 2024Review
- iPSCs chondrogenic differentiation for personalized regenerative medicine: a literature review.Stem cell research & therapy · 2024Review
- Article
- Streamlined, single-step non-viral CRISPR-Cas9 knockout strategy enhances gene editing efficiency in primary human chondrocyte populations.Arthritis research & therapy · 2024Article
- Regeneration of joint surface defects by transplantation of allogeneic cartilage: application of iPS cell-derived cartilage and immunogenicity.Inflammation and regeneration · 2023Review
- Clinical Potential of Cellular Material Sources in the Generation of iPSC-Based Products for the Regeneration of Articular Cartilage.International journal of molecular sciences · 2023Review
- Nutrient metabolism of the nucleus pulposus: A literature review.North American Spine Society journal · 2023Article
- Chondrogenic Differentiation of Human-Induced Pluripotent Stem Cells.Methods in molecular biology (Clifton, N.J.) · 2023Article
- Review
- Fluvastatin promotes chondrogenic differentiation of adipose-derived mesenchymal stem cells by inducing bone morphogenetic protein 2.BMC pharmacology & toxicology · 2022Article
54 more citing papers are in PubMed but not listed here.
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Authors and funding
10 authors at 4 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Gain-of-function mutations in the fibroblast growth factor receptor 3 gene (FGFR3) result in skeletal dysplasias, such as thanatophoric dysplasia and achondroplasia (ACH). The lack of disease models using human cells has hampered the identification of a clinically effective treatment for these diseases. Here we show that statin treatment can rescue patient-specific induced pluripotent stem cell (iPSC) models and a mouse model of FGFR3 skeletal dysplasia. We converted fibroblasts from thanatophoric dysplasia type I (TD1) and ACH patients into iPSCs. The chondrogenic differentiation of TD1 iPSCs and ACH iPSCs resulted in the formation of degraded cartilage. We found that statins could correct the degraded cartilage in both chondrogenically differentiated TD1 and ACH iPSCs. Treatment of ACH model mice with statin led to a significant recovery of bone growth. These results suggest that statins could represent a medical treatment for infants and children with TD1 and ACH.
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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.