CRISPR/Cas编辑猪用于个性化药物：不仅仅是临床前测试系统

Q3 Pharmacology, Toxicology and Pharmaceutics

Research Results in Pharmacology Pub Date : 2022-09-13 DOI:10.3897/rrpharmacology.8.83872

E. Leonova, Vasily V. Reshetnikov, J. Sopova

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引用次数: 0

摘要

基于crispr - cas的新型基因组编辑工具使得在猪基因组中引入各种精确的基因组修饰成为可能，包括同时引入多个编辑，将长DNA序列插入特定的靶向位点，以及进行核苷酸转换和翻转。猪作为一种重要的农业资源和生物医学研究的动物模型，具有优于其他模型的优势。猪在身体/器官大小、解剖学、生理学和代谢特征方面与人类有很高的相似性。猪的基因组可以被修改，携带与人类相同的基因突变，以复制遗传疾病，为药物的临床前试验提供支持。此外，基于crispr的猪抗原谱修饰可以提供猪器官用于异种移植，并且移植排斥反应最小。本文综述了内切酶介导的基因组编辑工具的最新进展，以及基因组编辑猪作为临床前试验的个性化测试系统和作为具有人类适合抗原谱的器官供体的研究进展。图形化的简介:

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CRISPR/Cas-edited pigs for personalized medicine: more than preclinical test-system

Novel CRISPR-Cas-based genome editing tools made it feasible to introduce a variety of precise genomic modifications in the pig genome, including introducing multiple edits simultaneously, inserting long DNA sequences into specifically targeted loci, and performing nucleotide transitions and transversions. Pigs serve as a vital agricultural resource and animal model in biomedical studies, given their advantages over the other models. Pigs share high similarities to humans regarding body/organ size, anatomy, physiology, and a metabolic profile. The pig genome can be modified to carry the same genetic mutations found in humans to replicate inherited diseases to provide preclinical trials of drugs. Moreover, CRISPR-based modification of pigs antigen profile makes it possible to offer porcine organs for xenotransplantation with minimal transplant rejection responses. This review summarizes recent advances in endonuclease-mediated genome editing tools and research progress of genome-edited pigs as personalized test-systems for preclinical trials and as donors of organs with human-fit antigen profile. Graphical abstract:

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