Daley George Q, Scadden David T
Division of Hematology/Oncology, Children's Hospital Boston, Boston, MA 02115, USA.
Cell. 2008 Feb 22;132(4):544-8. doi: 10.1016/j.cell.2008.02.009.
Resident pools of somatic stem cells in many organs are responsible for tissue maintenance and repair. The goal of regenerative medicine is to exploit these cells either by transplanting them from an exogenous source or by activating endogenous stem cells pharmacologically. For diseases caused by mutations in a single gene, the therapeutic goal is tissue replacement using stem cells engineered to correct the genetic defect. However, a number of technical hurdles must be overcome before therapies based on pluripotent human stem cells can enter the clinic.
许多器官中的体细胞干细胞库负责组织的维持和修复。再生医学的目标是通过从外源移植这些细胞或通过药物激活内源性干细胞来利用这些细胞。对于由单个基因突变引起的疾病，治疗目标是使用经过工程改造以纠正基因缺陷的干细胞进行组织替代。然而，在基于多能人类干细胞的疗法进入临床之前，必须克服许多技术障碍。
