Akademik Veri Yönetim Sistemi
UFUK 2020 Projesi, H2020 Diğer Kariyer Geliştirme Projeleri, 2020 - 2022
Osteogenesis Imperfecta (OI) is a prenatal onset debilitating inherited disorder and seen in 1 in 10.000-20.000 live birth. Affected children and adults suffer from multiple bone fractures causing severe bone deformities, requiring hospitalization and surgery. Severe type of OI (type III or severe type IV) cause life-long wheelchair boundary, spinal deformities and kyphoscoliosis predisposing them to respiratory morbidities and premature death. Currently, there is no curative treatment for OI and the only pharmaceutical treatments are palliative and fail to reduce fracture frequency and address the underlying bone brittleness and collagen defect.
However, novel fetal-derived mesenchymal stem cell (MSC) transplantation to OI patients is potentially decreasing severity of the disease with potential improvement of fracture rate and lengthwise growth. Treatment with fetal derived MSC in the prenatal or early postnatal period is believed to be more effective.
There is an ongoing EU Horizon 2020 funded project for fetal derived MSC transplantation in the host center, and the project includes a network of centers in the United Kingdom, the Netherlands and Germany. The Boost Brittle Bones Before Birth (BOOSTB4) clinical trial is investigating the safety and efficacy of this innovative therapy in OI.