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    • 41. 发明申请
    • CELL MEDIATED GENE DELIVERY USING MUSCLE DERIVED CELLS FOR TREATING MUSCLE-AND BONE-RELATED INJURY OR DYSFUNCTION
    • 使用肌肉衍生细胞进行细胞介导的基因交付,用于治疗肌肉和骨骼相关的伤害或功能障碍
    • WO9956785A3
    • 2001-04-19
    • PCT/US9909451
    • 1999-04-30
    • UNIV PITTSBURGH
    • CHANCELLOR MICHAEL BHUARD JOHNNY
    • A61K35/34A61K38/18A61K38/44A61K48/00A61P13/02A61P19/08A61P21/00C12N5/077C12N5/06C12N5/08C12N5/10
    • A61K35/34A61K48/00C12N5/0658C12N5/0659C12N2501/105C12N2501/113C12N2501/115C12N2501/13C12N2510/00C12N2510/04
    • The present invention provides muscle-derived cells, preferably myoblasts and muscle-derived stem cells, genetically engineered to contain and express one or more heterologous genes or functional segments of such genes, for delivery of the encoded gene products at or near sites of musculoskeletal, bone, ligament, meniscus, cartilage or genitourinary disease, injury, defect, or dysfunction. Ex vivo myoblast mediated gene delivery of human inducible nitric oxide synthase, and the resulting production of nitric oxide at and around the site of injury, are particularly provided by the invention as a treatment for lower genitourinary tract dysfunctions. Ex vivo gene transfer for the musculoskeletal system includes genes encoding acidic fibroblast growth factor, basic fibroblast growth factor, epidermal growth factor, insulin-like growth factor, platelet derived growth factor, transforming growth factor- beta , transforming growth factor- alpha , nerve growth factor and interleukin-1 receptor antagonist protein (IRAP), bone morphogenetic protein (BMPs), cartilage derived morphogenetic protein (CDMPs), vascular endothelial growth factor (VEGF), and sonic hedgehog proteins.
    • 本发明提供肌肉衍生的细胞,优选成肌细胞和来自肌肉的干细胞,其被遗传工程化以含有和表达这种基因的一个或多个异源基因或功能区段,用于在肌肉骨骼的位点或附近递送编码的基因产物, 骨,韧带,半月板,软骨或泌尿生殖系疾病,损伤,缺陷或功能障碍。 本发明特别提供离体成肌细胞介导的人诱导型一氧化氮合酶的基因递送以及所产生的损伤部位周围及其周围的一氧化氮,作为降低泌尿生殖道功能障碍的治疗。 肌肉骨骼系统的离体基因转移包括编码酸性成纤维细胞生长因子,碱性成纤维细胞生长因子,表皮生长因子,胰岛素样生长因子,血小板衍生生长因子,转化生长因子-β,转化生长因子-α,神经生长的基因 因子和白细胞介素-1受体拮抗蛋白(IRAP),骨形态发生蛋白(BMPs),软骨来源的形态发生蛋白(CDMPs),血管内皮生长因子(VEGF)和声音刺猬蛋白。