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Nat Chem Biol：科学家设计出新方法 助力疾病个体化疗法的开发

2016-12-13 佚名 生物谷

个体化疗法
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图片摘自：medcitynews.com 日前，一项刊登于国际杂志Nature Chemical Biology上的研究报告中，来自斯克利普斯研究所的研究人员通过研究开发出了一种新方法来设计新型个体化疗法，用来治疗由RNA引发的不治之症，细胞的RNA能够执行数千种必要的功能，但很多RNAs能够以不受控制的方式来发挥作用并且引发疾病。 数十年来，科学家们不断研究来开发靶向作用人类

图片摘自：medcitynews.com

日前，一项刊登于国际杂志Nature Chemical Biology上的研究报告中，来自斯克利普斯研究所的研究人员通过研究开发出了一种新方法来设计新型个体化疗法，用来治疗由RNA引发的不治之症，细胞的RNA能够执行数千种必要的功能，但很多RNAs能够以不受控制的方式来发挥作用并且引发疾病。

数十年来，科学家们不断研究来开发靶向作用人类机体RNAs的候选药物，但却困难重重，因为很多候选药物都不能达到预期的有效性和选择性，而且还会引发一定的副作用。这项研究中，研究者就开发了多种方法来克服目前所遇到的困难。研究者Disney表示，这项研究就像科幻小说一样，我们首次提出了多种方法来克服这些障碍；随着外科医生手术刀精确性的提高，我们就能够设计出很多小分子来寻找并且破坏引发疾病的RNAs。

此外研究者还开发出了新型的化学方法来利用诱发疾病的RNA作为药物合成的催化剂开发新型药物，这些研究对于治疗RNA疾病具有广泛的意义；强直性肌营养不良症1型是一种无法治愈的遗传性疾病，其是由一种名为“三联体重复”的RNA缺失引发的疾病，相比正常个体的遗传序列而言，患者机体中的基因三核苷酸会发生在重复序列的扩增；在很多遗传性疾病中都会出现两个拷贝的问题基因，突变的拷贝会诱发疾病，而正常的拷贝则会帮助细胞存活下去，在此之前研究者并不能够选择性地识别疾病的基因产物。

这项研究中，研究者设计出了小型分子来选择性地识别大型、和疾病相关的等位基因，研究者Suzanne G. Rzuczek表示，我们开发了多种方法能够制造出等位基因选择性的小分子，这种小分子仅仅能够选择性地寻找引发疾病的基因产物；所有方法都能够精准识别毒性的r(CUG)重复，更为重要的是，这种突变重复也是唯一的靶点；这项研究同时还提供了一种创新性方法，通过成像技术来追踪疾病细胞中RNA的运动。

最后研究者Lesley Colgan表示，本文中我们将化学方法同显微镜操作技术相结合，这种新型组合型方法能够强有力地帮助我们鉴别出新方法来对细胞中引发疾病的RNA进行追踪和处理。

原始出处

Suzanne G Rzuczek, Lesley A Colgan, Yoshio Nakai, Michael D Cameron, Denis Furling, Ryohei Yasuda & Matthew D Disney.Precise small molecule recognition of a toxic CUG RNA repeat expansion.Nat Chem Biol.2016

相关会议推荐

2017 疾病特异性模型研究及应用研讨会

会议时间：2017.03.03-2017.03.04     会议地点：上海

会议详情： http://www.bioon.com/z/2017iPSCs/

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    2017-04-07 liye789132251

    #Nat#

    0

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    2017-06-22 kord1982

    #新方法#

    0

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    2017-04-18 sunylz

    #Bio#

    0

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    2017-07-10 12498ebem31暂无昵称

    #Biol#

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    2017-08-12 yaanren

    #个体化疗法#

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    2016-12-15 zhouqu_8

    #个体化#

    0

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