{"title":"随心所欲CRISPR-碱基编辑产生用于免疫疗法的新型癌症限制性抗原","authors":"Ute E. Burkhardt, Edward F. Fritsch","doi":"10.1038/s41435-023-00227-6","DOIUrl":null,"url":null,"abstract":"","PeriodicalId":12691,"journal":{"name":"Genes and immunity","volume":"24 6","pages":"292-294"},"PeriodicalIF":5.0000,"publicationDate":"2023-12-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Serving up whatever you wish: CRISPR-base editing generates novel cancer-restricted antigens for immunotherapy\",\"authors\":\"Ute E. Burkhardt, Edward F. Fritsch\",\"doi\":\"10.1038/s41435-023-00227-6\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"\",\"PeriodicalId\":12691,\"journal\":{\"name\":\"Genes and immunity\",\"volume\":\"24 6\",\"pages\":\"292-294\"},\"PeriodicalIF\":5.0000,\"publicationDate\":\"2023-12-11\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Genes and immunity\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://www.nature.com/articles/s41435-023-00227-6\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"GENETICS & HEREDITY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Genes and immunity","FirstCategoryId":"3","ListUrlMain":"https://www.nature.com/articles/s41435-023-00227-6","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"GENETICS & HEREDITY","Score":null,"Total":0}
引用次数: 0
摘要
Casirati 等人的研究重点是急性髓性白血病（AML）标志物 FLT3、CD123 和 KIT，Wellhausen 等人的研究重点是原型造血系抗原 CD45。两个研究小组都生成了 CAR 构建物，并进行了表位图谱绘制，以确定各自的靶表位。然后，两组研究人员都转而使用 CRISPR 碱基编辑技术[6]引入非同义突变，这些突变分别是由于腺嘌呤碱基编辑器或胞嘧啶碱基编辑器产生的 A > G 或 C > T 转变，从而使 CAR T 细胞表位无效。CRISPR 碱基编辑避免了对 DNA 双股断裂/同源定向修复机制或供体 DNA 模板及其相关风险的依赖。只要选择正确的引导 RNA 和碱基编辑酶组合，就能在所有四个标记物中进行单个氨基酸替换，这种替换足以阻止相应 CAR T 细胞的识别，同时保持修饰后靶蛋白的功能。靶上编辑效率很高（79% 到 90% 以上），未编辑的细胞保留了未编辑靶标分子的表达，从而将它们扔到狼群中进行体内选择。虽然发现了脱靶效应和 "旁观者突变"，但影响极小，与传统化疗的基因毒性风险或疾病进展风险相比，Casirati 等人认为潜在危害很小。
期刊介绍：
Genes & Immunity emphasizes studies investigating how genetic, genomic and functional variations affect immune cells and the immune system, and associated processes in the regulation of health and disease. It further highlights articles on the transcriptional and posttranslational control of gene products involved in signaling pathways regulating immune cells, and protective and destructive immune responses.
京公网安备 11010802042870号
京ICP备2023020795号-1
分享
求助内容：
应助结果提醒方式：
扫码关注我们
