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作 者:化春晓 薛淑文 郭依琳 杨宇霞[1] 孔祥东[1] HUA Chunxiao;XUE Shuwen;GUO Yilin(Genetics and Prenatal Diagnosis Center,The First Affiliated Hospital of Zhengzhou University,Henan 450052,China)
机构地区:[1]郑州大学第一附属医院遗传与产前诊断中心,450052
出 处:《医学研究杂志》2023年第6期64-68,共5页Journal of Medical Research
基 金:河南省医学科技攻关计划项目(LHGJ20190075)。
摘 要:目的通过检测成人及胎儿体内是否存在SaCas9蛋白抗体,观察患者体内针对SaCas9蛋白抗体的体液免疫情况,从而进一步评价CRISPR/SaCas9系统临床应用的安全性并探究治疗遗传病的新方法。方法收集于郑州大学第一附属医院遗传与产前诊断中心进行唐氏筛查或产前诊断孕妇检测后欲废弃的血清、羊水、胎儿脐带血样本,通过酶联免疫吸附测定(enzyme linked immunosorbent assay,ELISA)法检测样本中SaCas9蛋白抗体的存在情况。结果在42例孕妇血清样本中均可检测到SaCas9蛋白抗体,18例胎儿羊水及10例胎儿脐带血样本中均未检测出。结论成人血清内存在SaCas9蛋白抗体,而羊水和脐血内均未检测出,表明胎儿体内可能没有针对CRISPR/SaCas9系统的体液免疫,从而为遗传病的宫内基因治疗提供了一定依据。Objective To evaluate the safety of clinical application of CRISPR/SaCas9 system and explore new methods for the treatment of genetic diseases by detecting the presence of SaCas9 protein antibodies in adults and fetuses,and observing the humoral immunity against SaCas9 protein antibodies.Methods Serum,amniotic fluid and fetal umbilical cord blood were collected from pregnant women′s samples,which were discarded samples for the tests of Down syndrome or prenatal diagnosis.Samples were detected by enzyme linked immunosorbent assay(ELISA)to test the presence of SaCas9 protein antibody.Results SaCas9 antibody was detected in serum samples of 42 pregnant women,but not in amniotic fluid of 18 fetuses and umbilical cord blood of 10 fetuses.Conclusion SaCas9 protein antibody was found in adult serum,but not in amniotic fluid and umbilical cord blood,indicating that there may be no humoral immunity against CRISPR/SaCas9 system in fetuses,thus providing a certain basis for intrauterine gene therapy for genetic diseases.
关 键 词:CRISPR/Cas9 遗传性疾病 SaCas9蛋白抗体 宫内基因治疗 体液免疫
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