干细胞在治疗学中的应用以及通过CRISPR Cas9系统进行位点特异性基因组编辑

Muhammad Javed Iqbal, Muhammad Mukheed, A. Khan, S. Irfan, Usama Hayat, Muhammad Umair Khalid, M. Raza, Kainat Amjad, Marriyam Tallat
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引用次数: 2

摘要

干细胞是一种未成熟的细胞,具有分化为体内所有特定的成熟细胞的能力。干细胞的两个主要特征是可自我再生和能够分化为所有成熟的、功能性的和成体的细胞类型。有两大类:a)多能干细胞,有可能分化成所有的成人细胞;b)多能干细胞,有能力分化成许多成人细胞,但不是所有的细胞类型。由于干细胞的自我再生能力,它被用于治疗、组织再生、疾病建模、再生药物和治疗心血管疾病、神经系统疾病如帕金森病,最重要的是用于治疗癌症。人类诱导多能干细胞能够分化成多种功能特异的成体细胞,为人类疾病的研究和治疗提供了良好的平台。利用CRISPR Cas9系统和TALENs等基因组编辑工具,通过诱导位点特异性突变、移框突变和缺失,在hPSCs中产生多种DNA变异。目前,CRISPR Cas9是一种更有效、更常用的基因组编辑方法,它来源于细菌细胞。
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Stem Cells Applications in Therapeutics and Site-Specific Genome Editing Through CRISPR Cas9 System
Stem cells are immature cells that have ability to differentiate into all specific and mature cells in body. The two main characteristics of stem cells are selfrenewable and ability to differentiate into all mature, functional and adult cells types. There are the two major classes a) pluripotent stem cells which have potential to differentiate in all adult cell and b) multipotent stem cells which have capacity to differentiate into many adult cells but not in all cell types. Due to the self-renewable ability stem cells are used in therapeutics, tissue regeneration, disease modeling, regenerative medicines and to treat cardiovascular diseases, neural disorders such as Parkinson’s disease and most importantly to treat carcinomas. The human induced pluripotent stem cells provide a great platform to study and treatment of human diseases because these are able to differentiate into many functional and specialized adult cells of body. The genome editing tools such as CRISPR Cas9 system and TALENs are used to generate multiple DNA variants in hPSCs by inducing site specific mutations, frame shift mutation and deletion. In present days CRISPR Cas9 is more efficient and frequent method for genome editing which is derived from bacterial cell.
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