γ-分泌酶调节剂在阿尔茨海默病治疗中的潜在应用。
Potential use of γ-secretase modulators in the treatment of Alzheimer disease.
作者信息
Wagner Steven L, Tanzi Rudolph E, Mobley William C, Galasko Douglas
机构信息
Department of Neurosciences, University of California, San Diego, School of Medicine, La Jolla, CA 92063, USA.
出版信息
Arch Neurol. 2012 Oct;69(10):1255-8. doi: 10.1001/archneurol.2012.540.
Abstract
Although significant progress has occurred in the past 20 years regarding our understanding of Alzheimer disease pathogenesis, we have yet to identify disease-modifying therapeutics capable of substantially altering the clinical course of this prevalent neurodegenerative disease. In this short review, we discuss 2 approaches that are currently being tested clinically (γ-secretase inhibition and γ-secretase modulation) and emphasize the significant differences between these 2 therapeutic approaches. We also discuss certain genetic- and biomarker-based translational and clinical trial paradigms that may assist in developing a useful therapeutic agent.
摘要
尽管在过去20年里，我们对阿尔茨海默病发病机制的理解取得了重大进展，但我们尚未找到能够显著改变这种常见神经退行性疾病临床进程的疾病修饰疗法。在这篇简短的综述中，我们讨论了目前正在进行临床测试的两种方法（γ-分泌酶抑制和γ-分泌酶调节），并强调了这两种治疗方法之间的显著差异。我们还讨论了某些基于基因和生物标志物的转化和临床试验模式，这些模式可能有助于开发出有效的治疗药物。
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