首页> 外国专利> RECOMBINANT AAV VECTORS EXPRESSING OSTEOPROTECTIVE GENES, INCLUDING HAS2 AND LUBRICIN, USEFUL IN THE TREATMENT OF OSTEOARTHRITIS AND RELATED JOINT CONDITIONS IN MAMMALS

RECOMBINANT AAV VECTORS EXPRESSING OSTEOPROTECTIVE GENES, INCLUDING HAS2 AND LUBRICIN, USEFUL IN THE TREATMENT OF OSTEOARTHRITIS AND RELATED JOINT CONDITIONS IN MAMMALS

机译:表达osteoprotective基因的重组AAV载体,包括HAS2和润滑素,可用于哺乳动物的骨关节炎和相关关节病的治疗。

摘要

The present disclosure relates to recombinant viral vectors, to pharmaceutical compositions comprising such recombinant vectors, and to methods for prevention and treatment of osteoarthritis in mammals. In particular, this disclosure provides adeno-associated virus (AAV) vectors capable of expressing, in a host, osteoprotective/chondroprotective bioactive proteins, including hyaluronan synthase 2 (HAS2) and lubricin (PRG4). Methods of production of these AAV are provided, as are methods of treatment of osteoarthritis in mammalian joints, by the long-term gene expression of osteoprotective/chondroprotective proteins, including HAS2 and PRG4, in both synovial and chondrocyte cells.
机译:本公开涉及重组病毒载体,涉及包含这种重组载体的药物组合物,以及涉及预防和治疗哺乳动物骨关节炎的方法。特别地,本公开提供了能够在宿主中表达包括透明质酸合酶2(HAS2)和lubricin(PRG4)的骨保护性/软骨保护性生物活性蛋白的腺相关病毒(AAV)载体。通过在滑膜和软骨细胞中长期保护骨/软骨保护蛋白(包括HAS2和PRG4)的基因表达,提供了产生这些AAV的方法以及治疗哺乳动物关节中的骨关节炎的方法。

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