Gafni Y, Turgeman G, Liebergal M, Pelled G, Gazit Z, Gazit D
Skeletal Biotech. Lab., Hebrew University-Hadassah Medical Center, Jerusalem.
Gene Ther. 2004 Feb;11(4):417-26. doi: 10.1038/sj.gt.3302197.
Adult stem cells reside in adult tissues and serve as the source for their specialized cells. In response to specific factors and signals, adult stem cells can differentiate and give rise to functional tissue specialized cells. Adult mesenchymal stem cells (MSCs) have the potential to differentiate into various mesenchymal lineages such as muscle, bone, cartilage, fat, tendon and ligaments. Adult MSCs can be relatively easily isolated from different tissues such as bone marrow, fat and muscle. Adult MSCs are also easy to manipulate and expand in vitro. It is these properties of adult MSCs that have made them the focus of cell-mediated gene therapy for skeletal tissue regeneration. Adult MSCs engineered to express various factors not only deliver them in vivo, but also respond to these factors and differentiate into skeletal specialized cells. This allows them to actively participate in the tissue regeneration process. In this review, we examine the recent achievements and developments in stem-cell-based gene therapy approaches and their applications to bone, cartilage, tendon and ligament tissues that are the current focus of orthopedic medicine.
成体干细胞存在于成体组织中，并作为其特化细胞的来源。响应特定的因子和信号，成体干细胞可以分化并产生功能性组织特化细胞。成人间充质干细胞（MSC）有潜力分化为各种间充质谱系，如肌肉、骨骼、软骨、脂肪、肌腱和韧带。成人间充质干细胞可以相对容易地从不同组织如骨髓、脂肪和肌肉中分离出来。成人间充质干细胞在体外也易于操作和扩增。正是成人间充质干细胞的这些特性使其成为骨骼组织再生的细胞介导基因治疗的焦点。经过基因工程改造以表达各种因子的成人间充质干细胞不仅在体内递送这些因子，还对这些因子作出反应并分化为骨骼特化细胞。这使它们能够积极参与组织再生过程。在本综述中，我们研究了基于干细胞的基因治疗方法的最新成果和进展，以及它们在骨科医学当前关注的骨、软骨、肌腱和韧带组织中的应用。
