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Gene discovery and validation for neurodegenerative diseases.

机译:神经退行性疾病的基因发现和验证。

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摘要

Treatment of neurodegenerative diseases, such as Alzheimer's disease, multiple sclerosis, Parkinson's disease and amyotrophic lateral sclerosis (ALS), represents a major challenge for the pharmaceutical industry. These disorders have common and unique molecular pathological characteristics that result in serious reductions in nervous-system functionality. Key to developing novel and efficacious therapeutics is the discovery of new gene targets. Genomic, proteomics and bioinformatic analyses are identifying vast amounts of genes whose expression is associated with the pathology of a specific disease. Extensive validation studies performed in parallel with drug development are crucial for the selection of appropriate target genes. This review outlines some of the current progress in gene discovery for neurodegenerative disease.
机译:神经退行性疾病的治疗,例如阿尔茨海默氏病,多发性硬化症,帕金森氏病和肌萎缩性侧索硬化症(ALS),是制药行业的主要挑战。这些疾病具有共同且独特的分子病理学特征,导致神经系统功能严重下降。开发新型有效疗法的关键是发现新的基因靶标。基因组学,蛋白质组学和生物信息学分析正在鉴定其表达与特定疾病的病理学相关的大量基因。与药物开发同时进行的广泛验证研究对于选择合适的靶基因至关重要。这篇综述概述了神经退行性疾病的基因发现方面的一些最新进展。

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