Osteogenesis Imperfecta
Osteogenesis imperfecta is a group of connective tissue disorders, a syndrome mainly characterized by heightened bone fragility. Our company stands at the forefront of rare disease research, extending comprehensive services, to the demands of researchers working for the innovative therapy of osteogenesis imperfecta.
Overview of Osteogenesis Imperfecta
Osteogenesis imperfecta is a rare genetic disorder characterized by brittle bones that break easily. With a suspected incidence of 1 in 20,000 individuals, osteogenesis imperfecta is a complex disorder that manifests in brittle bones prone to fractures, alongside various associated symptoms such as blue sclera, hearing impairment, and joint hypermobility.
Fig.1 Therapeutics in osteogenesis imperfecta studies to improve bone parameters. (Alcorta-Sevillano, N., et al., 2022)Pathogenesis of Osteogenesis Imperfecta
The underlying pathogenic mechanism of osteogenesis imperfecta is primarily linked to the impaired production of collagen type I, a crucial structural protein in bones. This deficiency leads to decreased bone mineral density and abnormal bone structure, contributing to the increased susceptibility to fractures.
Therapeutics of Osteogenesis Imperfecta
Small Molecule Drugs Therapy
Bisphosphonates (pamidronate, neridronate, or zoledronate) and synthetic parathyroid hormone (teriparatide) are used to increase bone mass density and prevent fractures.
Monoclonal Antibody Therapy
Denosumab (a monoclonal antibody against receptor RANKL) and anti-TGF-β antibodies (Fresolimumab) can affect osteoclasts to improve bone strength.
Cell Therapy
Stem cells transplantation, such as bone marrow / mesenchymal stem cell (BMSC) transplantation increased growth and mineral content and reduced fracture rate.
Gene Therapy
Genetic engineering techniques such as CRISPR / Cas9 provide a prospect for the therapy of osteogenesis imperfecta by potentially silencing or inactivating mutant genes.
Our Services
Our professional researchers and robust technical support form the bedrock of our services, providing a platform for animal model and therapeutics development, which empowers your deep dive into osteogenesis imperfecta pathogenesis and advancing innovative therapies.
Platforms of Osteogenesis Imperfecta Therapy Development
Animal Models of Osteogenesis Imperfecta
The utilization of animal models in osteogenesis imperfecta research represents a critical step in evaluating potential therapeutics and elucidating the underlying mechanisms of the disorder. Our company's commitment to providing animal models to support your research.
| Chemical-induced Models | ||
| Some chemicals can be used to induce bone fragility in animals, mimicking some aspects of the disease of osteogenesis imperfecta. | ||
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| Genetically Engineered Models | ||
| Genetic engineering models can induce osteogenesis imperfecta-like phenotypes in animals at specific developmental stages or in specific tissues by targeting genes involved in collagen synthesis and modification. | ||
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| Optional Species | Mice, Rats, Zebrafish, Non-Human Primates, Others | |
Our company with professional researchers and technical experts is committed to supporting your research of pharmacokinetics and biosafety evaluations. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.
References
- Alcorta-Sevillano, Natividad et al. "Murine Animal Models in Osteogenesis Imperfecta: The Quest for Improving the Quality of Life." International journal of molecular sciences 24.1 (2022): 184.
- Botor, Malwina et al. "Osteogenesis Imperfecta: Current and Prospective Therapies." Biomolecules 11.10 (2021): 1493.
- Etich, Julia et al. "Osteogenesis imperfecta-pathophysiology and therapeutic options." Molecular and cellular pediatrics 7.1 (2020): 9.
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.