病症小鼠模型20年的治疗发展:一个范围审查
Vanessa F Langness1, Danielle A Simmons1, Tyne L M McHugh1,2,3
1Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Palo Alto, California, USA.
概括
在小鼠模型中治疗病 (蛋白病理的疾病) 的治疗策略经常显示出希望,但面临翻译挑战. 小鼠和人类之间的治疗时间和终点评估的差异限制了临床成功.
科学领域:
- 神经科学是一个神经科学.
- 药理学 药理学是指药理学的学科.
- 遗传学 是一个遗传学.
背景情况:
- 病是一种神经退行性疾病,由异常的蛋白聚合和认知衰退来定义.
- 在小鼠模型中的MAPT (MAPT基因) 突变对于研究病症和评估潜在治疗方法至关重要.
- 相当多的研究已经利用这些模型来探索治疗干预措施.
研究的目的:
- 在MAPT (MAPT基因) 突变病症小鼠模型中测试的治疗干预措施的综合范围审查.
- 识别治疗策略的趋势,模型选择,管理途径和结果措施.
- 分析与疾病发病相关的治疗疗效和干预时间.
主要方法:
- 在MAPT小鼠模型中对409个治疗评估进行系统的搜索和审查.
- 分析治疗方法,使用的小鼠模型,输送方法和评估的终点.
- 对治疗效果的评估和与治疗开始的相关性,涉及病理.
主要成果:
- 许多经过测试的治疗方法在多个不同终点的多个MAPT小鼠模型中显示出积极的效果.
- 在临床前研究中,干预措施经常在tau病理发生之前或在tau病理发作时开始.
- 认知功能和突触退化等关键终点通常被低估,单性别研究是常见的.
结论:
- 尽管临床前取得了成功,但许多病疗法在人类临床试验中表现出有限的疗效.
- 治疗时间,终点评估,生物标志物使用和模型限制的差异构成了从小鼠到人类转换的障碍.
- 改善临床前研究设计,包括各种终点和生物标志物,对于推进病疗法至关重要.
关键词:
阿尔茨海默氏症 (AD) 是一种疾病.鼠标模型是MAPT的鼠标模型.前叶退行症 (FTLD) 是一种前叶退行症.范围审查 范围审查 范围审查突触突触突触是一个突触突触.病症是一种病症.治疗策略 治疗策略治疗治疗治疗治疗治疗治疗更多相关视频
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