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CRISPR/Cas9与多能干细胞相结合从嵌合体猪获取人类器官的潜力。

The potential of the combination of CRISPR/Cas9 and pluripotent stem cells to provide human organs from chimaeric pigs.

作者信息

Feng Wanyou, Dai Yifan, Mou Lisha, Cooper David K C, Shi Deshun, Cai Zhiming

机构信息

Shenzhen Key Laboratory of Xenotransplantaton, State and Local Joint Cancer Genome Clinical Application of Key Technology Laboratory, Shenzhen Second People's Hospital, First Affiliated Hospital of Shenzhen University, Shenzhen 518039, China.

Jiangsu Key Laboratory of Xenotransplantation, Nanjing Medical University, Nanjing 210029, China.

出版信息

Int J Mol Sci. 2015 Mar 23;16(3):6545-56. doi: 10.3390/ijms16036545.

Abstract

Clinical organ allotransplantation is limited by the availability of deceased human donors. However, the transplantation of human organs produced in other species would provide an unlimited number of organs. The pig has been identified as the most suitable source of organs for humans as organs of any size would be available. Genome editing by RNA-guided endonucleases, also known as clustered regularly interspaced short palindromic repeat (CRISPR/Cas9), in combination with induced pluripotent stem cells (iPSC), may have the potential to enable the creation of human organs from genetically-modified chimaeric pigs. These could potentially provide an unlimited supply of organs that would not be rejected by the recipient's immune system. However, substantial research is needed to prove that this approach will work. Genetic modification of chimaeric pigs could also provide useful models for developing therapies for various human diseases, especially in relation to drug development.

摘要

临床器官同种异体移植受到已故人类供体可用性的限制。然而,移植其他物种产生的人类器官将提供数量无限的器官。猪已被确定为最适合人类的器官来源,因为任何大小的器官都可获得。由RNA引导的核酸内切酶(也称为成簇规律间隔短回文重复序列,即CRISPR/Cas9)进行的基因组编辑,与诱导多能干细胞(iPSC)相结合,可能有潜力从基因改造的嵌合猪中培育出人类器官。这些器官有可能提供无限的供应,且不会被受体的免疫系统排斥。然而,需要大量研究来证明这种方法可行。嵌合猪的基因改造也可为开发各种人类疾病的治疗方法提供有用的模型,特别是在药物研发方面。

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