Synthetic DNA confers cell-specificity

IF 5.9 3区 农林科学 Q1 VETERINARY SCIENCES Lab Animal Pub Date : 2024-11-26 DOI:10.1038/s41684-024-01484-2
Alexandra Le Bras
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合成 DNA 赋予细胞特异性
顺式调节元件(CRE)是非编码 DNA 序列,通过与转录因子结合来调节附近基因的转录。CREs对于协调基因表达程序、驱动细胞特异性分化至关重要,因此在开发需要组织特异性的基因疗法方面具有巨大潜力。自然》(Nature)杂志上的一项研究报告了一种新方法的开发,该方法可设计出能够驱动细胞类型特异性转基因表达的合成 CREs。研究人员开发了这种方法,将可快速预测任何 DNA 序列活性的深度学习模型与可设计具有编程功能的新型 CRE 的模块化平台相结合。研究小组随后利用该模型设计了数千种具有程序特异性的合成 CRE,并应用于三种人类细胞系(红细胞前体、肝细胞和神经母细胞瘤)。接下来,他们进行了体外测试,结果表明合成序列在驱动细胞类型特异性基因表达方面优于天然序列。最后,他们在斑马鱼和小鼠胚胎中测试了合成的 CRE,证实了它们的组织特异性表达。这种方法对提高基因疗法的特异性很有价值:Gosai, S.J. et al. Nature 634, 1211-1220 (2024)
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来源期刊
Lab Animal
Lab Animal 农林科学-兽医学
CiteScore
0.60
自引率
2.90%
发文量
181
审稿时长
>36 weeks
期刊介绍: LabAnimal is a Nature Research journal dedicated to in vivo science and technology that improves our basic understanding and use of model organisms of human health and disease. In addition to basic research, methods and technologies, LabAnimal also covers important news, business and regulatory matters that impact the development and application of model organisms for preclinical research. LabAnimal's focus is on innovative in vivo methods, research and technology covering a wide range of model organisms. Our broad scope ensures that the work we publish reaches the widest possible audience. LabAnimal provides a rigorous and fair peer review of manuscripts, high standards for copyediting and production, and efficient publication.
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