Motor symptoms of Parkinson's disease (PD) are caused by degeneration and progressive loss of nigrostriatal dopamine neurons. Currently, no cure for this disease is available. Existing drugs alleviate PD symptoms but fail to halt neurodegeneration. Glial cell line–derived neurotrophic factor (GDNF) is able to protect and repair dopamine neurons in vitro and in animal models of PD, but the clinical use of GDNF is complicated by its pharmacokinetic properties. The present study aimed to evaluate the neuronal effects of a blood‐brain‐barrier penetrating small molecule GDNF receptor Rearranged in Transfection agonist, BT13, in the dopamine system.

中文翻译：

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在转染激动剂期间重新排列的胶质细胞系衍生的神经营养因子受体支持体外多巴胺神经元并增强体内多巴胺释放

帕金森病 (PD) 的运动症状是由黑质纹状体多巴胺神经元的退化和进行性丧失引起的。目前，还没有治愈这种疾病的方法。现有药物可缓解 PD 症状，但无法阻止神经变性。神经胶质细胞系衍生的神经营养因子 (GDNF) 能够在体外和 PD 动物模型中保护和修复多巴胺神经元，但 GDNF 的临床应用因其药代动力学特性而复杂化。本研究旨在评估在转染激动剂 BT13 中重新排列的血脑屏障穿透小分子 GDNF 受体在多巴胺系统中的神经元效应。