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线粒体DNA替代疗法研究现状
时间:2016-12-06 14:18:55  作者:editor  来源:Nature

  线粒体DNA替代疗法新技术将来自携带缺陷线粒体卵细胞的遗传物质转移到拥有健康线粒体的捐献者卵子内,产生的胚胎携带来自母亲和父亲的核DNA以及来自卵子捐献者的线粒体DNA,即所谓"一父两母"的人

  专家指出线粒体中特定的基因之间的差异可以解释有些基因复制的比别的快速的原因。卵母细胞的捐献者和病人最好具有相似的线粒体DNA以减少儿童时期变异DNA的竞争。这就导致了找与之匹配的捐赠者的难度加大。所以有科学家提出与其找与之匹配的捐赠者不如加大人力和物力去将“携带效应”减少甚至到零,这才是一个团队研究的意义。

  找到与母体线粒体匹配的捐赠者只是进行线粒体代替疗法的一个备选技术,并不是唯一技术。这不是完美的,这个技术或许是一条不通的路。人们在考虑治疗方法的时候必须明确的是这项技术并不是十分安全的。或许可以这么说,知道投入到临床实验时都有可能被别的技术所取代。

  在今年九月,纽约的一位妇产科医生乔恩张医生宣布,在墨西哥使用线粒体代替成功的避免了一个遗传性疾病—亚急性坏死脑病。医生指出这个刚出生的小男孩很健康,并且向HFEA(人类受精与胚胎管理局)提供了相关的报告。在这份报告里,被携带的变异基因并没有被敲除,而是被控制在9.2%以下。张医生希望这份报告尽快的被刊载出版,但是研究人员觉得还需再谨慎。

  Mitalipov’s study hints that specific genetic differences between mitochondria could explain why some replicate faster than others. Choosing egg donors and patients with otherwise similar mitochondrial DNA (or haplotypes) could reduce the chances of mutant mitochondria out-competing healthy counterparts in the cells of a developing child, says Iain Johnston, a biomathematician at the University of Birmingham, UK, who studies mitochondrial inheritance. “I would be more comfortable if these therapies were implemented with haplotype matching.”

  But Herbert is not convinced that Mitalipov’s explanation is correct and says that it might be difficult to find matching donors. “You’re setting up an arms race between the two mitochondrial genotypes and keeping your fingers crossed. I think a better use of time and money would be to redouble our efforts to get the carry-over as close to zero as we possibly can,” she says, an area of research her team is working on.

  The advisory report suggests that haplotype matching should be considered “as a precautionary step in donor selection”, but does not recommend it be mandatory. “It’s not perfect. There’s this chance of something going wrong,” says Robin Lovell-Badge, a developmental biologist at the Francis Crick Institute in London who was part of the HFEA panel. People considering the treatments, he adds, “must understand it’s impossible to ensure total safety until clinical trials have taken place”.

关键字:线粒体替代疗法
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