ArticleMolecular therapy : the journal of the American Society of Gene Therapy2012
Normal collagen and bone production by gene-targeted human osteogenesis imperfecta iPSCs.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 42 papers.
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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
42 citing papers in PubMed, 82 citations in OpenAlex.
- Inhibition of EED enhances osteogenic differentiation and bone formation: a potential therapeutic strategy for osteogenesis imperfecta.JBMR plus · 2026Article
- Proteomic alterations in patient bone-derived stromal cells and their secretomes in osteogenesis imperfecta.Molecular genetics and metabolism reports · 2026Article
- Current advances of bone homeostasis imbalance in the cause of hereditary metabolic bone diseases.EFORT open reviews · 2026Review
- Genetic landscape and phenotypic spectrum of osteogenesis imperfecta in the Kazakhstani pediatric population.Scientific reports · 2025Article
- Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases.Human gene therapy · 2024Review
- In Vitro Modelling of Osteogenesis Imperfecta with Patient-Derived Induced Mesenchymal Stem Cells.International journal of molecular sciences · 2024Article
- A Sporadic Case ofGenes · 2023Review
- Engineering considerations of iPSC-based personalized medicine.Biomaterials research · 2023Review
- Transcriptome and co-expression network analysis reveals the molecular mechanism of inosine monophosphate-specific deposition in chicken muscle.Frontiers in physiology · 2023Article
- Inhibitory Effect ofMedicina (Kaunas, Lithuania) · 2022Article
- Osteogenesis Imperfecta: Current and Prospective Therapies.Biomolecules · 2021Review
- Article
- Clinical Application of Bone Marrow Mesenchymal Stem/Stromal Cells to Repair Skeletal Tissue.International journal of molecular sciences · 2020Review
- Differentiation of Human Induced Pluripotent Stem Cells (hiPSCs) into Osteoclasts.Bio-protocol · 2020Article
- Versatility of Induced Pluripotent Stem Cells (iPSCs) for Improving the Knowledge on Musculoskeletal Diseases.International journal of molecular sciences · 2020Review
- Genomic Medicine: Lessons Learned From Monogenic and Complex Bone Disorders.Frontiers in endocrinology · 2020Review
- Human Fibroblasts as a Model for the Study of Bone Disorders.Frontiers in endocrinology · 2020Review
- Tissue engineered bone mimetics to study bone disorders ex vivo: Role of bioinspired materials.Biomaterials · 2019Review
- Low Osteogenic Yield in Human Pluripotent Stem Cells Associates with Differential Neural Crest Promoter Methylation.Stem cells (Dayton, Ohio) · 2018Article
- Generation and Applications of Induced Pluripotent Stem Cell-Derived Mesenchymal Stem Cells.Stem cells international · 2018Review
Corrections and comments
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Authors and funding
7 authors at 1 institution in 1 country.
Funding
Abstract
Osteogenesis imperfecta (OI) is caused by dominant mutations in the type I collagen genes. In principle, the skeletal abnormalities of OI could be treated by transplantation of patient-specific, bone-forming cells that no longer express the mutant gene. Here, we develop this approach by isolating mesenchymal cells from OI patients, inactivating their mutant collagen genes by adeno-associated virus (AAV)-mediated gene targeting, and deriving induced pluripotent stem cells (iPSCs) that were expanded and differentiated into mesenchymal stem cells (iMSCs). Gene-targeted iMSCs produced normal collagen and formed bone in vivo, but were less senescent and proliferated more than bone-derived MSCs. To generate iPSCs that would be more appropriate for clinical use, the reprogramming and selectable marker transgenes were removed by Cre recombinase. These results demonstrate that the combination of gene targeting and iPSC derivation can be used to produce potentially therapeutic cells from patients with genetic disease.
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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.