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默克的基础基因组编辑技术获得澳大利亚CRISPR切口酶专利

2018-08-16 MedSci MedSci

充满活力的科技公司默克(Merck)今天宣布,澳大利亚专利局(Australian Patent Office)已经批准了该公司的配对规律成簇的间隔短回文重复(CRISPR)切口酶使用专利申请。配对切口酶是提高安全性的有效措施,通过高度灵活且有效的脱靶效应降低方法推动特异性。这可提高 CRISPR 固定患病基因同时不影响健康基因的能力。默克执行董事会成员兼生命科学首席执行官吴博达(Udit Bat

充满活力的科技公司默克(Merck)今天宣布,澳大利亚专利局(Australian Patent Office)已经批准了该公司的配对规律成簇的间隔短回文重复(CRISPR)切口酶使用专利申请。配对切口酶是提高安全性的有效措施,通过高度灵活且有效的脱靶效应降低方法推动特异性。这可提高 CRISPR 固定患病基因同时不影响健康基因的能力。

默克执行董事会成员兼生命科学首席执行官吴博达(Udit Batra)表示:“最近数年,我们在发展 CRISPR 技术方面取得了巨大进展。现在是科学研究的关键时刻。默克的配对切口酶 CRISPR 技术对于为难治疾病开发疗法时需要高精度方法的研究人员而言很重要。这项新获得的专利代表着 CRISPR 疗法安全性的显著提升。”

此次获批的专利申请涉及基础 CRISPR 战略,即两个 CRISPR 切口酶目标共同基因靶向,并通过剪切或切割染色体序列的对应链造成双链断裂而通力合作。这项过程可按照与默克获得专利的 CRISPR 整合技术相同的方式任意选择包括外源或供体序列的植入。两个 CRISPR 绑定活动的要求大大减少了基因组其它地方的脱靶剪切机会。

除了批准配对切口酶专利申请之外,澳大利亚专利局最近还宣布在撤回四个独立、匿名提交的反对意见之后正式授予默克2017 CRISPR 整合专利。

默克的 CRISPR 整合专利组合包括在澳大利亚、加拿大、中国、欧洲、以色列、新加坡和韩国获得的专利。这些 CRISPR 专利涉及染色体整合,或切割真核细胞染色体序列,并将外部 DNA 序列植入真核细胞,以便实现想要的基因组变动。

配对 CRISPR 切口酶方法建基于默克 CRISPR 专利组合(包括 CRISPR 整合)的其它技术。商业机构如果想要纠正基因治疗患者体细胞的遗传缺陷,则需要默克基于 CRISPR 的 DNA 植入的知识产权。默克将为所有领域提供这个专利组合授权。

过去14年,默克高度致力于基因组编辑创新。该公司知道基因组编辑为生物研究与医学带来了重大进步。同时,日益提高的基因组编辑技术潜力获得了科学、法律和社会关注。默克既是基因组编辑技术的使用者,也是该技术的供应商,慎重考虑道德和法律标准,通过基因组编辑为研究提供支持。默克已经组建了生物伦理顾问小组,为默克开展的研究提供指导,包括基因组编辑的研究及应用。默克的生物伦理顾问小组已经确定了清晰的经营定位,在考虑科学和社会问题的同时,不会妨碍任何有前景的治疗方法的研究和应用。

默克是第一家在全球范围内为基因组编辑提供定制生物分子(TargeTron™ RNA 引导性 II 型内含子和 CompoZr™ 锌指核酸酶)的公司,推动全球研究者采用这些技术。默克还是第一家制造覆盖整个人类基因组的成簇 CRISPR 序列库的公司,让科学家能够探究更多关于疾病根源的问题,由此加快治愈疾病的进程。

除了基础基因组编辑研究之外,默克还为基因与细胞疗法开发提供支持,并制造病毒载体。2016年,默克推出了目标通过专业团队和更强资源推进新模式研究 -- 从基因组编辑到基因医学制造 -- 的基因组编辑计划,进一步巩固该公司致力于该领域的承诺。

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    2018-08-20 哈哈869

    血压

    0

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    2018-08-19 哈哈869

    学习了

    0

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    2018-08-18 哈哈869

    学习了

    0

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    2018-08-18 yuandd
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    2018-08-17 哈哈869

    学了

    0

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    2018-08-16 哈哈869

    学习了

    0

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如果你明白“CRISPR 介导基因编辑”这个词的含义,那么你一定知道我们现在能更加有效、快速且廉价地改造 DNA。这不可避免地带来了对基因治疗的严肃讨论,该技术通过直接修改某人的 DNA 来治疗遗传性疾病,如镰状细胞贫血症或血友病。你也可能听说过有目标的基因增强,以实现一个健康人提高其基因组品质的梦想。今天,美国国家科学院(the US National Academy of Science)

PNAS:在小鼠细胞中利用CRISPR-Cas9技术高效进行基因组编辑

为了利用CRISPR-Cas9基因编辑系统切割基因,人们必需设计一种与靶基因的DNA相匹配的RNA序列。大多数基因具有上百个这样的在基因组中的活性和独特性上存在差异的序列。因此,寻找最佳的序列很难通过手工实现。一种新的“CrispRGold”程序有助科学家们鉴定出最为高效的和最为特异性的RNA序列。这种程序是由德国马克斯-德尔布吕克分子医学中心科学家Klaus Rajewsky教授领导的一个研究团

三篇Nature文章揭示CRISPR/Cas9基因组编辑取得重大进展

大多数人类遗传病是由于点突变---DNA序列上的单个碱基错误---导致的。然而,当前的基因组编辑方法不能够高效地校正细胞中的这些突变,而且经常导致随机的核苷酸插入或删除(insertions or deletion, indel)。 如今,在一项新的研究中,来自美国哈佛大学的研究人员对CRISPR/Cas9技术进行改进,构建出一种新的“碱基编辑器(base editor)”,并且避免

JACS:上海科技大学季泉江组研发出“超级细菌”基因组编辑新技术

日前,国际期刊《Journal of the American Chemical Society》杂志上在线发表了上海科技大学物质学院(材料生物学研究部)季泉江助理教授课题组在人类致病菌金黄色葡萄球菌(包括其中的“超级细菌”)中首次建立起基于CRISPR/Cas9系统的、高效快速的基因组编辑方法。研究成果题为“Rapid and Efficient Genome Editing in Staphy

Prenat Diagn:围产医学2016年的显著进展

2016年的最新发现围绕着基因组编辑、线粒体替代、产前遗传学筛查和检测,以及胎儿超声领域的进展,还包括有创产前诊疗、寨卡病毒和巨细胞病毒的相关研究。基因编辑与线粒体替代基因编辑自2015年末发生的一系列事件对我们理解早期胚胎发育、不孕症治疗及单基因病(特别是线粒体病)的预防具有重要影响。2015年12月1日至3日,在美国国家科学院、伦敦皇家学会、中国科学院共同主持的人类基因编辑国际峰会上,建议

Nat Biotechnol:“基因魔剪”准确性低于预期

英国《自然·生物技术》杂志 16 日 在线发表 了一项重要研究:有“基因魔剪”之称的 CRISPR-Cas9 基因组编辑技术,在靶点附近引起的 DNA 删除或重排,比科学家此前预期得要严重。该发现意味着,研究人员必须密切观察基于 CRISPR-Cas9 疗法对编辑后细胞造成的序列变化。

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