Drug Development for Neurodegenerative Diseases
Drug Development for Neurodegenerative Diseases
复制标题
神经退行性疾病药物开发
DOI：
10.1007/978-4-431-54541-5_9
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发表时间：
2015
期刊：
影响因子：
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通讯作者：
Yoshitaka Nagai and Eiko N. Minakawa
中科院分区：
文献类型：
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作者：
Hideki Tsukahara;Yuya Nakamura;Takuya Murakami;Misako Endo,Yoshinobu Watanabe; Shinano Yu;Masaki Hara;Masatomo Mihara;Tatsuo Shimizu,Michiyasu Inoue; Yoshiyuki Marsuoka;Tsutomu Asano;Hiromichi Gotoh;Yoshikazu Goto.;Yoshitaka Nagai and Eiko N. Minakawa
神经退行性疾病，如阿尔茨海默病、帕金森病、肌萎缩性侧索硬化和多聚谷氨酰胺病，已被定义为一组难治性疾病，其特征在于大脑各个区域的神经元的进行性变性，导致神经和精神症状。分子遗传学和生物学研究表明，大多数神经退行性疾病是由蛋白质错误折叠和聚集引起的，因此它们被认为是所谓的蛋白质错误折叠疾病。此外，最近出现的证据表明，在这些疾病中形成的错误折叠的蛋白质聚集体具有相似的内在特征，即，它们通过朊病毒样感染机制繁殖。因此，正在广泛探索针对蛋白质错误折叠和聚集的各种治疗策略。在这里，我们介绍了新兴的疾病修饰治疗方法对神经退行性疾病，特别是那些针对有毒蛋白质的错误折叠和聚集。在不久的将来，人们迫切期待开发通常对广泛的神经退行性疾病有效的抗错误折叠和抗聚集剂。
Neurodegenerative diseases, such as Alzheimer’s disease, Parkinson’s disease, amyotrophic lateral sclerosis, and the polyglutamine diseases, have been defined as a group of intractable disorders, which are characterized by the progressive degeneration of neurons in various regions of the brain, resulting in neurological and psychiatric symptoms. Molecular genetics and biological studies have revealed that most neurodegenerative diseases are caused by protein misfolding and aggregation, and hence they are considered to belong to the so-called protein misfolding diseases. Moreover, recent emerging evidence has suggested that the misfolded protein aggregates formed in these diseases have similar intrinsic characteristics, i.e., they are propagated by prion-like infectious mechanisms. Therefore, various therapeutic strategies targeting protein misfolding and aggregation are being extensively explored. Here we introduce emerging disease-modifying therapeutic approaches against neurodegenerative diseases, particularly those targeting the misfolding and aggregation of toxic proteins. The development of anti-misfolding and anti-aggregation agents that are commonly effective against a wide range of neurodegenerative diseases is eagerly anticipated in the near future.