ArticleGene therapy2005
Correction of a mineralization defect by overexpression of a wild-type cDNA for COL1A1 in marrow stromal cells (MSCs) from a patient with osteogenesis imperfecta: a strategy for rescuing mutations that produce dominant-negative protein defects.
Article in Gene therapy, 2005. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 29 papers.
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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.
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Who cites it
29 citing papers in PubMed, 60 citations in OpenAlex.
- The double whammy of ER-retention and dominant-negative effects in numerous autosomal dominant diseases: significance in disease mechanisms and therapy.Journal of biomedical science · 2024Review
- Mesenchymal stem cells in the treatment of osteogenesis imperfecta.Cell regeneration (London, England) · 2023Review
- A recombinant adenoviral vector with a specific tropism to CD4-positive cells: a new tool for HIV-1 inhibition.Drug delivery and translational research · 2022Article
- Ponicidin Treatment Improved the Cell Proliferation, Differentiation, and Calcium Mineralization on the Osteoblast-Like MG-63 Cells.Applied biochemistry and biotechnology · 2022Article
- Ginsenoside Rc Promotes Bone Formation in Ovariectomy-Induced Osteoporosis In Vivo and Osteogenic Differentiation In Vitro.International journal of molecular sciences · 2022Article
- Augmenting and directing long-range CRISPR-mediated activation in human cells.Nature methods · 2021Article
- Clinical Application of Bone Marrow Mesenchymal Stem/Stromal Cells to Repair Skeletal Tissue.International journal of molecular sciences · 2020Review
- Effects of ginsenosides on bone remodelling for novel drug applications: a review.Chinese medicine · 2020Review
- Ginsenoside Rg1 promotes osteogenic differentiation of rBMSCs and healing of rat tibial fractures through regulation of GR-dependent BMP-2/SMAD signaling.Scientific reports · 2016Article
- Autologous implantation of BMP2-expressing dermal fibroblasts to improve bone mineral density and architecture in rabbit long bones.Journal of orthopaedic research : official publication of the Orthopaedic Research Society · 2015Article
- Research using Mesenchymal Stem/Stromal Cells: quality metric towards developing a reference material.Cytotherapy · 2015Review
- Effect of Intra-Medullar and Intra-Venous Infusions of Mesenchymal Stem Cells on Cell Engraftment byOrthopedic & muscular system : current research · 2014Article
- Molecular mechanisms of mesenchymal stem cell differentiation towards osteoblasts.World journal of stem cells · 2013Review
- Recommendations and considerations for the use of biologics in orthopedic surgery.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2012Review
- Gene therapy: implications for craniofacial regeneration.The Journal of craniofacial surgery · 2012Review
- Article
- Drosha regulates hMSCs cell cycle progression through a miRNA independent mechanism.The international journal of biochemistry & cell biology · 2011Article
- Plasmid-encapsulated polyethylene glycol-grafted polyethylenimine nanoparticles for gene delivery into rat mesenchymal stem cells.International journal of nanomedicine · 2011Article
- Management of Liver Failure: From Transplantation to Cell-Based Therapy.Cell medicine · 2011Article
- Stem cells and regenerative medicine: accomplishments to date and future promise.Therapeutic delivery · 2010Review
Corrections and comments
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Authors and funding
5 authors at 4 institutions in 1 country.
Funding
Abstract
Gene therapy for dominant-negative disorders presents a more difficult challenge than gene therapy for recessive disorders, since even partial replacement of a protein for a recessive disorder can reverse symptoms. Osteogenesis imperfecta (OI) has frequently served as a model disorder for dominant-negative defects of structural proteins. The disease is caused by mutations in type I collagen (COL1A1), the major structural component of bone, skin and other connective tissues. The severity of the phenotype is largely dependent on the ratio of normal to mutant type I procollagen synthesized by cells. Recently, attempts have been made to develop strategies for cell and gene therapies using the adult stem cells from bone marrow referred to as mesenchymal stem cells or marrow stromal cells (MSCs). In this study, we used MSCs from a patient with type III OI who was heterozygous for an IVS 41A+4C mutation in COL1A1. A hybrid genomic / cDNA construct of COL1A1 was transfected into the MSCs and the transfectants were expanded over a 200-fold. Transfected MSCs showed increased expression of the wild-type mRNA and protein. In vitro assays demonstrated that the transfected cells more efficiently differentiated into mineralizing cells. The results indicated that it is possible to overexpress COL1A1 cDNA in OI MSCs and thereby to correct partially the dominant-negative protein defect.
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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.