Pinjala Poojitha, Tryphena Kamatham Pushpa, Prasad Renuka, Khatri Dharmendra Kumar, Sun Woong, Singh Shashi Bala, Gugulothu Dalapathi, Srivastava Saurabh, Vora Lalitkumar
Molecular and Cellular Neuroscience Lab, Department of Pharmacology and Toxicology, National Institute of Pharmaceutical Education and Research (NIPER)-Hyderabad, Telangana-500037, Hyderabad, India.
Department of Anatomy, Korea University College of Medicine, Moonsuk Medical Research Building, 73 Inchon-Ro, Seongbuk-Gu, Seoul, 12841, Republic of Korea.
Biomater Res. 2023 May 16;27(1):46. doi: 10.1186/s40824-023-00381-y.
Since its discovery in 2012, CRISPR Cas9 has been tried as a direct treatment approach to correct the causative gene mutation and establish animal models in neurodegenerative disorders. Since no strategy developed until now could completely cure Parkinson's disease (PD), neuroscientists aspire to use gene editing technology, especially CRISPR/Cas9, to induce a permanent correction in genetic PD patients expressing mutated genes. Over the years, our understanding of stem cell biology has improved. Scientists have developed personalized cell therapy using CRISPR/Cas9 to edit embryonic and patient-derived stem cells ex-vivo. This review details the importance of CRISPR/Cas9-based stem cell therapy in Parkinson's disease in developing PD disease models and developing therapeutic strategies after elucidating the possible pathophysiological mechanisms.
自2012年被发现以来，CRISPR Cas9已被尝试作为一种直接治疗方法，用于纠正致病基因突变并建立神经退行性疾病的动物模型。由于迄今为止开发的任何策略都无法完全治愈帕金森病（PD），神经科学家渴望使用基因编辑技术，尤其是CRISPR/Cas9，对表达突变基因的遗传性PD患者进行永久性矫正。多年来，我们对干细胞生物学的理解有所提高。科学家们已开发出使用CRISPR/Cas9的个性化细胞疗法，用于在体外编辑胚胎干细胞和患者来源的干细胞。这篇综述详细阐述了基于CRISPR/Cas9的干细胞疗法在帕金森病中的重要性，即在阐明可能的病理生理机制后，开发PD疾病模型和治疗策略。
Zotero 插件
