Regenerative medicine for congenital skeletal diseases: Mesenchymal stem cell transplantation for hypophosphatasia

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  • 先天性骨系統疾患に対する再生医療：低ホスファターゼ症に対する間葉系幹細胞移植

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Abstract

<p>Congenital skeletal diseases (CSDs) can be diagnosed early by advances in diagnostic imaging and identification of causative genes. However, many patients have a fatal course or severely impaired daily activities. Therefore, therapeutic improvements are expected. To establish curative and permanent therapy for one of CSDs, hypophosphatasia (HPP), we are trying to achieve bone regeneration using mesenchymal stem cells (MSCs), which bring about bone and cartilage regeneration. So far, we succeeded in not only recovery of bone mineralization but also improvement of muscle weakness, respiratory disturbance and mental retardation for fatal HPP patients using allogeneic MSC transplantation, resulting in improvement in both prognosis and quality of life. But, normal bone architecture has not been restored. In the near future, we plan a new cell transplantation using highly-purified MSCs (rapidly expanded cells; REC), which have excellent proliferative ability and bone differentiation. Bone regenerative therapy using MSCs can be expected to be applied to curative treatments for patients with CSDs including HPP if these methodologies are established.</p>

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