Chen Yiju, Carter Richard L, Cho In K, Chan Anthony W S
Division of Neuropharmacology and Neurologic Diseases, Yerkes National Primate Research Center, 954 Gatewood Rd, NE Atlanta, GA 30329, USA; Department of Human Genetics, Emory University School of Medicine, 615 Michael St, Atlanta, GA 30322, USA.
Division of Neuropharmacology and Neurologic Diseases, Yerkes National Primate Research Center, 954 Gatewood Rd, NE Atlanta, GA 30329, USA; Department of Human Genetics, Emory University School of Medicine, 615 Michael St, Atlanta, GA 30322, USA; Genetics and Molecular Biology Program, Laney Graduate School, Emory University, 201 Dowman Drive, Atlanta, GA 30322, USA.
Drug Discov Today. 2014 Jul;19(7):980-4. doi: 10.1016/j.drudis.2014.02.012. Epub 2014 Mar 12.
Cell-based therapies are a viable option for the long-term treatment of Huntington's disease (HD), which is characterized by progressive neurodegeneration predominately in the striatum and cortex. Current research focuses on genetic suppression of the mutant huntingtin (mHTT) gene and cell replacement therapy of the lost cells in HD. As we discuss here, the recent development of induced pluripotent stem (iPS) cells technology demonstrated the potential of cell-based therapy in rodent models. It was shown that iPSCs were capable of differentiating into lost neurons in HD and stem cell grafts can improve motor deficiency in HD rodent models. Altogether, these findings have shown great promise for developing the foundation of the cell-based therapy.
基于细胞的疗法是治疗亨廷顿舞蹈症（HD）的一种可行的长期选择，该疾病的特征是主要在纹状体和皮质中进行性神经变性。目前的研究集中在对突变亨廷顿蛋白（mHTT）基因的基因抑制以及HD中丢失细胞的细胞替代疗法。正如我们在此讨论的，诱导多能干细胞（iPS）技术的最新进展证明了基于细胞的疗法在啮齿动物模型中的潜力。研究表明，iPSC能够分化为HD中丢失的神经元，并且干细胞移植可以改善HD啮齿动物模型中的运动缺陷。总之，这些发现为发展基于细胞的疗法奠定了基础，显示出巨大的前景。
