CRISPR/Cas9辅助干细胞治疗帕金森病

IF 11.3 1区 医学 Q1 Medicine Biomaterials Research Pub Date : 2023-05-16 DOI:10.1186/s40824-023-00381-y
Poojitha Pinjala, Kamatham Pushpa Tryphena, Renuka Prasad, Dharmendra Kumar Khatri, Woong Sun, Shashi Bala Singh, Dalapathi Gugulothu, Saurabh Srivastava, Lalitkumar Vora
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引用次数: 7

摘要

自2012年被发现以来,CRISPR Cas9一直被尝试作为纠正致病基因突变和建立神经退行性疾病动物模型的直接治疗方法。由于到目前为止还没有开发出能够完全治愈帕金森病(PD)的策略,神经科学家渴望使用基因编辑技术,特别是CRISPR/Cas9,来诱导表达突变基因的遗传性PD患者的永久纠正。多年来,我们对干细胞生物学的理解有所提高。科学家们利用CRISPR/Cas9在体外编辑胚胎和患者来源的干细胞,开发出了个性化的细胞疗法。本文在阐明可能的病理生理机制后,详细介绍了CRISPR/ cas9干细胞治疗帕金森病在建立PD疾病模型和制定治疗策略中的重要性。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

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CRISPR/Cas9 assisted stem cell therapy in Parkinson's disease.

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.

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来源期刊
Biomaterials Research
Biomaterials Research Medicine-Medicine (miscellaneous)
CiteScore
10.20
自引率
3.50%
发文量
63
审稿时长
30 days
期刊介绍: Biomaterials Research, the official journal of the Korean Society for Biomaterials, is an open-access interdisciplinary publication that focuses on all aspects of biomaterials research. The journal covers a wide range of topics including novel biomaterials, advanced techniques for biomaterial synthesis and fabrication, and their application in biomedical fields. Specific areas of interest include functional biomaterials, drug and gene delivery systems, tissue engineering, nanomedicine, nano/micro-biotechnology, bio-imaging, regenerative medicine, medical devices, 3D printing, and stem cell research. By exploring these research areas, Biomaterials Research aims to provide valuable insights and promote advancements in the biomaterials field.
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