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基因编辑技术介入胚胎发育 成功切除导致先天心脏病基因

2017-08-04 12:30:03   央广网

央广网北京8月4日消息(记者张奥实习记者于洋)据经济之声《天下公司》报道,英国《自然》杂志公布了一篇基因编辑的论文,美国和韩国科学家利用基因编辑技术删除了一颗人类胚胎中的变异基因,这种基因变异与一种遗传性心脏疾病有关,能够造成猝死和心力衰竭。如今科学家团队可以将变异部分在胚胎阶段删除,以此证明在生命早期通过编辑生殖细胞的DNA安全有效。

关于基因编辑crispr技术,《天下公司》节目曾在《烧脑季》中专门请过清华大学的谢震博士来讲解过。据谢震介绍:“这种系统是非常神奇的,核酸酶,也就是核酸DNA的剪刀,能够在小的RNA的指导下,结合在RNA和DNA互补配对的序列上。当互补序列配对后,剪刀就被定位到那个位置,就可以对那个位置进行双链的断裂的剪切,这样就会造成基因的断裂,我们也可以对基因进行修补或者替换。Crispr系统来源于细菌和古细菌,是一种自然进化的结果,任何一个细菌中都有它的天敌,它的天敌叫噬菌体——细菌的病毒。”

结合杂志上所讲的案例,谢震博士介绍了这种导致先天性心脏病的病原基因。

谢震说:“这个基因叫做MYBPC3,它与心肌肥大有关,显性遗传基变如果有突变,就会造成后代产生疾病心肌肥大,心肌肥大会造成左心室一定程度供血不足,逐渐地往外泵血能力就变差了。例如有些运动员的非正常死亡。这种疾病突变的频率是很高的,据报道,每500人中就有一人携带这种基因,所以这是比较常见的疾病突变。”

之前在《烧脑季》中讲到过,成年人疑难病症的基因剪辑,但是胚胎很小,也是生命体的最早期,这如何发现并且剪辑突变的基因?

谢震说:“这个过程中,男性病人的精子是携带有基因的,女性的卵子里是不携带的,所以当男性的精子受精过程中就把坏的基因拷贝到卵子里,Crisprcas技术靶向是把这个基因切掉。因为来源于母亲的基因是好的,它把基因又重新copy,把坏的修复掉。”

目前从这个试验的个例来看,不仅成功而且安全,那它广泛应用还有什么障碍?

谢震表示:“现在大家更关注的是编辑胚胎在伦理上有没有问题。这还是很有争议的。有科学家反对对胚胎编辑,原因有两个:第一,这种技术在成熟之前还存在一些风险,基因的剪刀可能剪切到好的基因上,这样就会造成一些不必要的突变,这些突变都是可遗传的。潜在的技术风险有可能带来的危害可能会比治疗疾病本身要大,这需要更多的研究。

第二,对于采取体外受精的病人,也可以筛查到底有哪些受精卵携带有坏的基因。这时候就可以把这些受精卵抛弃掉,停止发育。也可以把好的挑选出来,不对胚胎做编辑也可以使后代不用遗传下去。”

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