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广州医科大学人类胚胎基因修饰引起关注
诸平
2016年4月6日,《辅助生殖与遗传学杂志》(Journal of Assisted Reproduction and Genetics)刊登了广州医科大学附属第三医院范勇(Yong Fan,是此项研究论文的通讯作者)领导的研究团队所完成的一项有关人类胚胎基因编辑方面的研究——Xiangjin Kang , Wenyin He, Yuling Huang, Qian Yu, Yaoyong Chen, Xingcheng Gao, Xiaofang Sun, Yong Fan. Introducing precise genetic modifications into human 3PN embryos by CRISPR/Cas-mediated genome editing, Journal of Assisted Reproduction and Genetics (First online: 06 April 2016). DOI:10.1007/s10815-016-0710-8
此项让全球科学家的神经再一次被挑动(也可以留意:https://www.altmetric.com/details/6556977 )。MedicalXpress网站4月11日(当地时间)报道之后,到北京时间4月12日21点,其分享已经超过3000次(据观察大约每10分钟会增加50次分享)。国内由饶毅、鲁白、谢宇三位学者创办的移动新媒体平台——《知识分子》也有相关报道。
根据研究者介绍,他们进行此项研究的目的是对CRISPR技术在早期人类胚胎的精准基因编辑方面的应用进行评估并制定原则,从而为未来遗传性疾病的治疗提供了可能。
从2014年4月至9月,研究者从87名志愿者那里收集了213枚三原核受精卵,即原本不能正常发育的人类胚胎细胞。研究者通过基因编辑技术CRISPR,对这些受精卵中的基因ccr5进行编辑,结果发现26个人体胚胎细胞中,仅有4个细胞的基因成功被修饰,基因编辑的脱靶问题突出,表明该技术存在大量瓶颈。此次实验的所有胚胎在三天后均被销毁。研究者之所以选择该基因,是因为部分人群如果携带有ccr5突变基因,那么他们则拥有抵抗HIV病毒的能力,该突变基因能改变CCR5蛋白,使其能够阻止HIV病毒对人体免疫细胞的入侵。更多信息请浏览:
Chineseteam uses CRISPR to genetically modify human embryo
Xiangjin Kang , Wenyin He, Yuling Huang,Qian Yu, Yaoyong Chen, Xingcheng Gao, Xiaofang Sun, Yong Fan. Introducingprecise genetic modifications into human 3PN embryos by CRISPR/Cas-mediatedgenome editing, Journal of Assisted Reproduction and Genetics (Firstonline: 06 April 2016).DOI:10.1007/s10815-016-0710-8
Abstract
As a powerful technology for genome engineering, the CRISPR/Cas system has been successfully applied to modify the genomes of various species. The purpose of this study was to evaluate the technology and establish principles for the introduction of precise genetic modifications in early human embryos.
3PN zygotes were injected with Cas9 messenger RNA (mRNA) (100 ng/μl) and guide RNA (gRNA) (50 ng/μl). For oligo-injections, donor oligo-1 (99 bp) or oligo-2 (99 bp) (100 ng/μl) or dsDonor (1 kb) was mixed with Cas9 mRNA (100 ng/μl) and gRNA (50 ng/μl) and injected into the embryos.
By co-injecting Cas9 mRNA, gRNAs, and donor DNA, we successfully introduced the naturally occurring CCR5Δ32 allele into early human 3PN embryos. In the embryos containing the engineeredCCR5Δ32 allele, however, the other alleles at the same locus could not be fully controlled because they either remained wild type or contained indel mutations.
This work has implications for the development of therapeutic treatments of genetic disorders, and it demonstrates that significant technical issues remain to be addressed. We advocate preventing any application of genome editing on the human germline until after a rigorous and thorough evaluation and discussion are undertaken by the global research and ethics communities.
也可以留意:https://www.altmetric.com/details/6556977
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