生物谷报道：本周Science报道首次将人骨髓间质干细胞采用基因打靶方法，采用腺病毒相关病毒作为载体，将一个胶原基因COL1A1的dominant-negative mutant导入细胞，从而成功治愈了骨发育不良疾病。这是首次采用成体干细胞的基因打靶方法进行治疗，这一成果为今后成体干细胞的应用打开了新的窗口。
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.
Original article:
Gene Targeting in Stem Cells from Individuals with Osteogenesis Imperfecta
Joel R. Chamberlain, Ulrike Schwarze, Pei-Rong Wang, Roli K. Hirata, Kurt D. Hankenson, James M. Pace, Robert A. Underwood, Kit M. Song, Michael Sussman, Peter H. Byers, and David W. Russell
Science Feb 20 2004: 1198-1201. [Full Text] [PDF] [Supporting Online Material]
