Department of Molecular Medicine, Center for Regenerative Medicine, The Scripps Research Institute , La Jolla, California.
Stem Cells Dev. 2018 Jul 15;27(14):958-959. doi: 10.1089/scd.2018.0107. Epub 2018 Jun 18.
In 2012, we planned a program to develop a neuron replacement therapy for Parkinson's disease (PD) that would have the greatest promise to help the patients. PD is a movement disorder caused by the progressive, inevitable loss of a specific type of dopamine neuron in the brain. The only viable treatment to reverse the progress of the disease is to replace those neurons; we decided to make dopamine neurons that matched the patients, by differentiating induced pluripotent stem cells that we generated from individuals with PD. This autologous cell therapy is entering the regulatory approval process this year with the U.S. Food and Drug Administration to begin to transplant the cells in the following 1 to 2 years.
2012 年，我们计划开发一种治疗帕金森病（PD）的神经元替代疗法，这种疗法最有希望帮助患者。PD 是一种运动障碍，由大脑中特定类型的多巴胺神经元进行性、不可避免的丧失引起。唯一可行的治疗方法是逆转疾病的进展，通过分化我们从 PD 患者中生成的诱导多能干细胞来替代这些神经元；我们决定制造与患者匹配的多巴胺神经元。这种自体细胞疗法今年正在接受美国食品和药物管理局的监管批准程序，以便在未来 1 到 2 年内开始移植这些细胞。
