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Safety of intra-articular transplantation of lentivirally transduced mesenchymal stromal cells for haemophilic arthropathy in a non-human primate.

Int J Hematol. 2018; 
Ohmori T, Mizukami H, Katakai Y, Kawai S, Nakamura H, Inoue M, Shu T, Sugimoto H, Sakata Y.
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Codon Optimization … We synthesized two codon-optimized sequences using two different companies [Thermo Fisher Scientific (Waltham, MA) and GenScript (Piscataway, NJ)] with the aim of selecting the best sequence to express FVIII, because different algorisms may influence protein expression … Get A Quote

摘要

Joint bleeding and resultant arthropathy are major determinants of quality of life in haemophilia patients. We previously developed a mesenchymal stromal cell (MSC)-based treatment approach for haemophilic arthropathy in a mouse model of haemophilia A. Here, we evaluated the long-term safety of intra-articular injection of lentivirally transduced autologous MSCs in non-human primates. Autologous bone-marrow-derived MSCs transduced with a lentiviral vector expressing coagulation factor VIII (FVIII) were injected into the left knee joint of cynomolgus monkeys. We first conducted codon optimization to increase FVIII production in the cells. Lentiviral transduction of autologous MSCs resulted in a significant incre... More

關鍵詞

Arthropathy; Cell therapy; Haemophilia; Lentiviral vector; Mesenchymal stromal cells
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