生物谷報(bào)道：本周Science報(bào)道首次將人骨髓間質(zhì)干細(xì)胞采用基因打靶方法,，采用腺病毒相關(guān)病毒作為載體，將一個(gè)膠原基因COL1A1的dominant-negative mutant導(dǎo)入細(xì)胞,，從而成功治愈了骨發(fā)育不良疾病,。這是首次采用成體干細(xì)胞的基因打靶方法進(jìn)行治療，這一成果為今后成體干細(xì)胞的應(yīng)用打開了新的窗口,。
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]
