基因组编辑技术能够精确靶向修饰生物体基因组特定位点、人为改造生物遗传信息。自21世纪初,基因组编辑技术得到迅猛发展,锌指核酸酶(zinc finger nucleases, ZFN)、转录激活样受体因子(transcription- activating-like receptor factor, TALEN)和成簇的规律间隔短回文重复序列及其相关系统(clustered regularly interspaced short palindromic repeats/Cas endonucleases, CRISPR/Cas)3种新型基因组编辑系统先后诞生。基因组编辑技术能对生物体内源基因进行精确靶向修饰,被广泛应用于生物医学研究领域。猪与人类亲缘关系接近,在生理特征与病理病程等诸多方面与人类具有相似性,是人类疾病动物模型与异种器官移植研究的重要对象。本文主要介绍了ZFN、TALEN和CRISPR/Cas9 3种基因组编辑技术的发展概况与作用机理,综述了基因编辑猪在人类疾病模型、器官移植与人源化器官培育等生物医学领域里的最新研究进展,并对基因编辑猪的应用前景进行了展望。

Genome editing technologies (GETs) can precisely alter the genomic sequences and modify the genetic information at the target site of an organism. Since the beginning of the 21st century, the GETs, including zinc finger nucleases (ZFN), transcription-activating-like receptor factor (TALEN), and clustered regularly interspaced short palindromic repeats/Cas endonucleases (CRISPR/Cas), have been successively developed. The GETs can easily engineer the targeted genomic site of animals to exhibit a desired phenotype(s), thereby providing valuable tools in biomedical research. The pigs are closely related to human, in terms of similarities in physiological properties and pathogenic characters. Thus, pigs have been used as important animal models in studies of human disease, xenotransplantation, and humanized organs regeneration. In this review, we summarize the development of the three GETs, research progress of genome-edited pigs as disease models and organ donors for xenotransplantation, and the prospects of their applications in future biomedical research.