Gene targeting in stem cells from individuals with osteogenesis imperfecta
Gene targeting in stem cells from individuals with osteogenesis imperfecta
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DOI：
10.1126/science.1088757
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发表时间：
2004-02-20
期刊：
影响因子：
56.9
通讯作者：
Russell, DW
中科院分区：
文献类型：
--
作者：
Chamberlain, JR;Schwarze, U;Russell, DW
成体干细胞通过体外遗传操作和自体移植的结合，提供了治疗许多疾病的潜力。间充质干细胞（MSCs，也称为骨髓基质细胞）是成体干细胞，可以从骨骼中分离出增殖的贴壁细胞。MSCs可以分化成多种细胞类型，存在于多种组织中，包括骨、脂肪、软骨和肌肉，这使它们成为各种基于细胞的治疗的理想候选者。在这里，我们使用腺相关病毒载体破坏来自脆性骨紊乱成骨不全症个体的MSCs中的显性阴性突变COL1A1胶原基因，证明在成人干细胞中成功靶向基因。
Adult stem cells offer the potential to treat many diseases through a combination of ex vivo genetic manipulation and autologous transplantation. Mesenchymal stem cells (MSCs, also referred to as marrow stromal cells) are adult stem cells that can be isolated as proliferating, adherent cells from bones. MSCs can differentiate into multiple cell types present in several tissues, including bone, fat, cartilage, and muscle, making them ideal candidates for a variety of cell-based therapies. Here, we have used adeno-associated virus vectors to disrupt dominant-negative mutant COL1A1 collagen genes in MSCs from individuals with the brittle bone disorder osteogenesis imperfecta, demonstrating successful gene targeting in adult human stem cells.