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Science：美国基因编辑先驱张锋发表新工具：编辑RNA可治疗多种顽疾

2017-10-26 佚名 科学网

基因编辑编辑RNA治疗多种顽疾
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作为首位将基因编辑系统CRISPR用于哺乳动物细胞的开拓者，美国博德研究所华人科学家张锋近来对RNA编辑产生了兴趣，一月内连发两篇相关论文在顶级学术期刊。

作为首位将基因编辑系统CRISPR用于哺乳动物细胞的开拓者，美国博德研究所华人科学家张锋近来对RNA编辑产生了兴趣，一月内连发两篇相关论文在顶级学术期刊。

美国东部时间10月25日，国际学术期刊《科学》发表了张锋团队一篇介绍CRISPR新系统“REPAIR”的文章。“REPAIR”的基本元件是一种取名为PspCas13b的酶和ADAR2蛋白。“REPAIR”可高效地修复RNA的单个核苷，因不会改变DNA信息而更为安全，将为基础研究和临床治疗提供一个新的工具。

Cas13酶家族最近受张锋团队青睐。10月4日，在发表于《自然》期刊的论文上，张锋团队证实了另一种酶Cas13a能够在哺乳动物细胞中特异性地下调内源性RNA和报告RNA的水平。

“基因编辑的首要目标是矫正导致疾病的突变。现在，我们已经很擅长让基因失活，但要修复已丢失的蛋白质功能则更具挑战得多。编辑RNA这一新技能打开了更多的可能，几乎可以在所有细胞中修复蛋白质功能，有助于多种疾病治疗。”张锋表示。

和此前CRISPR系统用于编辑DNA不同，张锋团队在普雷沃氏细菌（Prevotella）中找到了PspCas13b酶。这是Cas13酶家族中能使RNA失去活性的“佼佼者”，是潜在的RNA“剪刀”。

但张锋团队赋予PspCas13b的“使命”不是去让RNA失活，相反的，他们设计了PspCas13b的“变体”。这个“变体”失去了“剪刀”的功能，但会牢牢地结合在特定的RNA片段上。同时，PspCas13b“变体”的搭档——ADAR2蛋白会将该片段上的腺嘌呤核苷（A）替换成次黄嘌呤核苷（I）。

为何要做此替换？原来，鸟嘌呤核苷（G）突变为腺嘌呤核苷（A）时有发生，而这被认为和杜氏肌营养不良症、帕金森病等疾病密切相关。

人类的很多疾病信息都编码在DNA这个“生命脚本”上。基因编辑技术的出现使得科学家有了修改DNA的可能，给治愈疾病带来希望。但由于基因承载着生命最根源的信息，对DNA进行编辑有着安全和伦理上的顾虑。
RNA编辑有所不同。RNA是以DNA单链为模板的遗传信息载体。DNA发出 “错误指令”，经RNA转录，翻译给蛋白质并执行功能，才有疾病的表现。若中途拦截，矫正RNA上的错误信息，让蛋白质接收到正确的信息，可起到治疗效果。

DNA与RNA之间的关系

安全和灵活是“REPAIR”的优势。“REPAIR无需篡改基因组就可以修复突变。而且RNA会自动降解，对它的修改或是可逆的。”张锋实验室学生、该论文的共同第一作者David Cox表示。

为了让“REPAIR”更加成熟，张锋团队对其进行了优化，设计出“REPAIR2”版本，将在转录组中可检测到的脱靶次数从1.8万次降至20次。据介绍，“REPAIR2”对目标RNA的编辑效率为20%-40%，最高达到51%。

为验证“REPAIR”系统在疾病治疗上的潜力，张锋团队人工合成了会造成范科尼贫血和X连锁性肾源性尿崩症的突变，并将这些突变引至人体细胞中，最后成功通过“REPAIR”在RNA层次上修复了致病突变。

接下来，张锋团队表示将继续从自然界寻找线索，设计其他附件，适用于更多类型的核苷酸修复。

原始出处：
David B. T. Cox, Jonathan S. Gootenberg, Omar O. Abudayyeh, et al.RNA editing with CRISPR-Cas13.Science.25 Oct 2017.DOI: 10.1126/science.aaq0180

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    2017-10-28 jichang

    #SCIE#

    0

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    2017-10-27 xuexin53

    学习

    0

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    2017-10-27 戒馋，懒，贪

    谢谢分享.学习了

    0

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    2017-10-27 jihuaijun1112

    学习学习学习

    0

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    2017-10-26 1e127dd4m40(暂无匿称)

    厉害!!

    0

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昨日，大洋彼岸的美国传来喜讯。知名华人学者，CRISPR-Cas9基因编辑技术的先驱之一张锋教授与Emmanuelle Charpentier教授、Jennifer Doudna教授、Luciano Marraffini教授、以及Francisco J.M. Mojica教授一道，喜获全球知名的阿尔伯尼生物医学奖（Albany Medical Center Prize in Medicine an

基因编辑技术先驱之一张锋 喜获阿尔伯尼生物医学奖

今日，大洋彼岸的美国传来喜讯。知名华人学者，CRISPR-Cas9基因编辑技术的先驱之一张锋教授与Emmanuelle Charpentier教授、Jennifer Doudna教授、Luciano Marraffini教授、以及Francisco J.M. Mojica教授一道，喜获全球知名的阿尔伯尼生物医学奖（Albany Medical Center Prize in Medicine an

基因编辑临床应用引争议：现存三大问题亟待解决

本月，先是科学家们宣布成功修正人胚胎中肥厚型心肌病致病基因，后有世界首批经基因编辑对器官移植无“毒”的小猪诞生；上个月，美国一个专家委员会以１０比０的投票，建议食品和药物管理局批准第一种癌症基因疗法……在新一代基因编辑工具尤其是ＣＲＩＳＰＲ推动下，新型基因疗法正加速迈向临床应用。四大热点值得关注基因编辑在多个领域拥有巨大的应用潜力，其中一大热门是治疗遗传病，尤其是单基因遗传病。据估计，大概有一万种

深度报告：基因编辑改写人类的未来

基因编辑编年史可以追溯到30年前，第一次基因编辑是在酵母细胞实验中完成。但是直到过去五年，随着CRISPR技术出现并且迅速成为目前最受欢迎的基因编辑手段，诞生于上世纪70年代末的重组DNA技术才真正被视为在人类新药研发中取得了革命性进步。

基因编辑小猪“见证”中国科研竞争力提升

近日，世界首批内源性逆转录病毒灭活猪诞生，从根本上解决了猪器官用于人体移植的异种病毒传播风险，对未来人类健康有着重要意义。