Al-Khayri Jameel M, Ravindran Mamtha, Banadka Akshatha, Vandana Chendanda Devaiah, Priya Kushalva, Nagella Praveen, Kukkemane Kowshik
Department of Agricultural Biotechnology, College of Agriculture and Food Sciences, King Faisal University, Al-Ahsa 31982, Saudi Arabia.
Department of Biotechnology and Genetics, School of Sciences, JAIN (Deemed-to-be-University), Bangalore 560027, India.
Pharmaceuticals (Basel). 2024 Oct 18;17(10):1391. doi: 10.3390/ph17101391.
Amyotrophic Lateral Sclerosis (ALS) is a severe neurodegenerative disorder marked by the gradual loss of motor neurons, leading to significant disability and eventual death. Despite ongoing research, there are still limited treatment options, underscoring the need for a deeper understanding of the disease's complex mechanisms and the identification of new therapeutic targets. This review provides a thorough examination of ALS, covering its epidemiology, pathology, and clinical features. It investigates the key molecular mechanisms, such as protein aggregation, neuroinflammation, oxidative stress, and excitotoxicity that contribute to motor neuron degeneration. The role of biomarkers is highlighted for their importance in early diagnosis and disease monitoring. Additionally, the review explores emerging therapeutic approaches, including inhibitors of protein aggregation, neuroinflammation modulators, antioxidant therapies, gene therapy, and stem cell-based treatments. The advantages and challenges of these strategies are discussed, with an emphasis on the potential for precision medicine to tailor treatments to individual patient needs. Overall, this review aims to provide a comprehensive overview of the current state of ALS research and suggest future directions for developing effective therapies.
肌萎缩侧索硬化症（ALS）是一种严重的神经退行性疾病，其特征是运动神经元逐渐丧失，导致严重残疾并最终死亡。尽管研究不断，但治疗选择仍然有限，这凸显了深入了解该疾病复杂机制并确定新治疗靶点的必要性。本综述对ALS进行了全面审视，涵盖其流行病学、病理学和临床特征。它研究了导致运动神经元变性的关键分子机制，如蛋白质聚集、神经炎症、氧化应激和兴奋性毒性。强调了生物标志物在早期诊断和疾病监测中的重要作用。此外，本综述探讨了新兴的治疗方法，包括蛋白质聚集抑制剂、神经炎症调节剂、抗氧化疗法、基因治疗和基于干细胞的治疗。讨论了这些策略的优势和挑战，重点是精准医学根据个体患者需求定制治疗的潜力。总体而言，本综述旨在全面概述ALS研究的现状，并为开发有效疗法提出未来方向。
