Ryberg Henrik, Bowser Robert
Department of Pathology, Center for ALS Research, University of Pittsburgh School of Medicine, Pittsburgh, PA 15261, USA.
Expert Rev Proteomics. 2008 Apr;5(2):249-62. doi: 10.1586/14789450.5.2.249.
Amyotrophic lateral sclerosis (ALS) is a progressive motor neuron disease with largely unknown pathogenesis that typically results in death within a few years from diagnosis. There are currently no effective therapies for ALS. Clinical diagnosis usually takes several months to complete and the long delay between symptom onset and diagnosis limits the possibilities for effective intervention and clinical trials. The establishment of protein biomarkers for ALS may aid an earlier diagnosis, facilitating the search for effective therapeutic interventions and monitoring drug efficacy during clinical trials. Biomarkers could also be used to discriminate between subtypes of ALS, to measure disease progression and to detect susceptibility for developing ALS or monitor adverse effects of drug treatment. The present review will discuss the opportunities and proteomic platforms used for biomarker discovery efforts in ALS, summarizing putative ALS protein biomarkers identified in different biofluids.
肌萎缩侧索硬化症(ALS)是一种进行性运动神经元疾病,其发病机制大多未知,通常在确诊后几年内导致死亡。目前尚无针对ALS的有效疗法。临床诊断通常需要数月才能完成,症状出现与诊断之间的长时间延迟限制了有效干预和临床试验的可能性。建立ALS的蛋白质生物标志物可能有助于早期诊断,便于寻找有效的治疗干预措施并在临床试验期间监测药物疗效。生物标志物还可用于区分ALS的亚型、测量疾病进展以及检测患ALS的易感性或监测药物治疗的不良反应。本综述将讨论用于ALS生物标志物发现工作的机会和蛋白质组学平台,总结在不同生物流体中鉴定出的假定ALS蛋白质生物标志物。