Katta Madhumitha, Mathew Blessy Aksa, Chaturvedi Pragya, Ludhiadch Abhilash, Munshi Anjana
Complex Disease Genomics and Precision Medicine Laboratory, Department of Human Genetics and Molecular Medicine, Central University of Punjab, Bathinda, 151401, India.
Neurol Sci. 2023 Jan;44(1):19-36. doi: 10.1007/s10072-022-06356-6. Epub 2022 Sep 6.
Neurological diseases (NDs) are one of the leading causes of disability and the second leading cause of death globally. Among these stroke, Alzheimer's disease (AD), and Parkinson's disease (PD) are the most common NDs. A rise in the absolute number of individuals affected with these diseases indicates that the current treatment strategies in management and prevention of these debilitating diseases are not effective sufficiently. Therefore, novel treatment strategies are being explored to cure these diseases by addressing the causative mechanisms at the molecular level. Advanced therapies like gene therapy (gene editing and gene silencing) and stem cell therapies aim to cure diseases by gene editing, gene silencing and tissue regeneration, respectively. Gene editing results in the deletion of the aberrant gene or insertion of the corrected gene which can be executed using the CRISPR/Cas gene editing tool a promising treatment strategy being explored for many other prevalent diseases. Gene silencing using siRNA silences the gene by inhibiting protein translation, thereby silencing its expression. Stem cell therapy aims to regenerate damaged cells or tissues because of their ability to divide into any type of cell in the human body. Among these approaches, gene editing and gene silencing have currently been applied in vitro and to animal models, while stem cell therapy has reached the clinical trial stage for the treatment of NDs. The current status of these strategies suggests a promising outcome in their clinical translation.
神经疾病（NDs）是导致残疾的主要原因之一，也是全球第二大死因。在这些疾病中，中风、阿尔茨海默病（AD）和帕金森病（PD）是最常见的神经疾病。受这些疾病影响的个体绝对数量的增加表明，目前在管理和预防这些使人衰弱的疾病方面的治疗策略不够有效。因此，正在探索新的治疗策略，通过在分子水平上解决致病机制来治愈这些疾病。基因治疗（基因编辑和基因沉默）和干细胞治疗等先进疗法分别旨在通过基因编辑、基因沉默和组织再生来治愈疾病。基因编辑导致异常基因的缺失或校正基因的插入，这可以使用CRISPR/Cas基因编辑工具来实现，这是一种正在为许多其他常见疾病探索的有前景的治疗策略。使用小干扰RNA（siRNA）的基因沉默通过抑制蛋白质翻译来使基因沉默，从而使其表达沉默。干细胞治疗旨在再生受损细胞或组织，因为它们能够分化为人体中的任何类型的细胞。在这些方法中，基因编辑和基因沉默目前已应用于体外和动物模型，而干细胞治疗已进入治疗神经疾病的临床试验阶段。这些策略的现状表明它们在临床转化方面有一个有前景的结果。
