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Review Article

帕金森氏病的基因治疗：GDNF有望成为有前景的治疗策略

卷 20, 期 3, 2020

页: [207 - 222] 页: 16

弟呕挨: 10.2174/1566523220999200817164051

价格: $65

摘要

帕金森氏病（一种常见的神经退行性疾病）中常规治疗方法的局限性导致了另一种基因治疗方法的发展。建立了针对多巴胺能神经元再生，与多巴胺合成有关的酶的产生，丘脑下核神经元，星形胶质细胞和小胶质细胞的调节以及增强神经营养因子的多种治疗选择。基于病毒载体的多巴胺传递，前药途径，胎儿腹侧中脑组织移植和编码基因传递的多巴胺合成酶是重要的疗法，许多试验已明确支持这种疗法。这篇综述主要阐述了神经胶质细胞源性神经营养因子在减轻帕金森氏病的运动症状和多巴胺能神经元丧失中的重要作用。 GDNF的神经保护和神经再生作用是通过临床前和临床研究成果确定的。 GDNF家族配体与相关受体的结合导致酪氨酸激酶家族的受体-配体复合物激活Ret受体的形成，该受体仅在多巴胺能神经元中表达，通过与必需蛋白的结合在帕金森氏病中起重要作用编码的基因。此外，该综述建立了递送方面，例如重组GDNF的心室递送，使用输注导管的实质内和肠内递送。综述重点介绍了GDNF递送的问题和挑战，以及克服这些问题的基本措施，例如基因治疗组合，递送载体的优化，更新的靶向设备，抑制集中超声技术的运动症状，修改患者选择标准以及开发新的基于递送的策略通过脂质体和包囊细胞的使用，促进神经营养因子的安全有效递送，并为患者建立常规治疗方法。

关键词: 神经退行性疾病，多巴胺能神经元再生，丘脑下核神经元，病毒载体，胎儿腹中中脑，神经胶质细胞系衍生的神经营养因子，视网膜受体。

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