Urinary biomarkers for amyotrophic lateral sclerosis: candidates, opportunities and considerations.
Urinary biomarkers for amyotrophic lateral sclerosis: candidates, opportunities and considerations.
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DOI：
10.1093/braincomms/fcad287
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发表时间：
2023
影响因子：
4.8
通讯作者：
中科院分区：
文献类型：
--
作者：
肌萎缩侧索硬化症是一种无情的神经退行性疾病，大多数在3-5年内致命，并根据进行性上下运动神经元变性的证据进行诊断。大约15%的肌萎缩侧索硬化症患者也有额颞叶变性，基因突变占10%。肌萎缩侧索硬化症是一种可变的异质性疾病，越来越清楚的是，许多不同的疾病过程最终导致运动神经元的变性。有一个深刻的需要清楚地阐明和衡量病理过程中发生的。需要这些信息来根据个体的病理指纹为患有肌萎缩侧索硬化症的个体定制治疗。对于新的候选疗法，还需要根据预期的治疗结果选择患者并测量治疗成功与否的方法。生物标志物是满足这些需求的重要工具，尿液是候选生物流体生物标志物的丰富来源。本综述将描述肌萎缩侧索硬化症的有前途的候选尿液生物标志物和其他可能的尿液候选物在未来的调查领域以及尿液生物标志物的局限性。为肌萎缩侧索硬化症寻找客观的基于液体的生物标志物，反映表型异质性疾病中发生的病理过程，对于评估治疗是否有效至关重要。这需要评估替代的非侵入性生物流体，如尿液，并注意标准化收集，处理和验证候选人。
Amyotrophic lateral sclerosis is a relentless neurodegenerative disease that is mostly fatal within 3–5 years and is diagnosed on evidence of progressive upper and lower motor neuron degeneration. Around 15% of those with amyotrophic lateral sclerosis also have frontotemporal degeneration, and gene mutations account for ∼10%. Amyotrophic lateral sclerosis is a variable heterogeneous disease, and it is becoming increasingly clear that numerous different disease processes culminate in the final degeneration of motor neurons. There is a profound need to clearly articulate and measure pathological process that occurs. Such information is needed to tailor treatments to individuals with amyotrophic lateral sclerosis according to an individual’s pathological fingerprint. For new candidate therapies, there is also a need for methods to select patients according to expected treatment outcomes and measure the success, or not, of treatments. Biomarkers are essential tools to fulfil these needs, and urine is a rich source for candidate biofluid biomarkers. This review will describe promising candidate urinary biomarkers of amyotrophic lateral sclerosis and other possible urinary candidates in future areas of investigation as well as the limitations of urinary biomarkers. Finding objective fluid-based biomarkers for amyotrophic lateral sclerosis that reflect the pathological processes occurring in a phenotypically heterogeneous disease is essential for assessing if treatments are working or not. This requires evaluating alternate non-invasive biofluids such as urine, with caveats such as standardizing collection, processing and validating candidates.
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影响因子：
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