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Synthetic Virus-Derived Nanosystems (SVNs) for Delivery and Precision Docking of Large Multifunctional DNA Circuitry in Mammalian Cells.
Pharmaceutics ( IF 5.4 ) Pub Date : 2020-08-11 , DOI: 10.3390/pharmaceutics12080759
Francesco Aulicino 1 , Julien Capin 1 , Imre Berger 1, 2
Affiliation  

DNA delivery is at the forefront of current research efforts in gene therapy and synthetic biology. Viral vectors have traditionally dominated the field; however, nonviral delivery systems are increasingly gaining traction. Baculoviruses are arthropod-specific viruses that can be easily engineered and repurposed to accommodate and deliver large sequences of exogenous DNA into mammalian cells, tissues, or ultimately organisms. These synthetic virus-derived nanosystems (SVNs) are safe, readily customized, and can be manufactured at scale. By implementing clustered regularly interspaced palindromic repeats (CRISPR) associated protein (CRISPR/Cas) modalities into this system, we developed SVNs capable of inserting complex DNAs into genomes, at base pair precision. We anticipate a major role for SVNs as an attractive alternative to viral vectors in accelerating genome engineering and gene therapy applications in the future.

中文翻译:

合成病毒衍生的纳米系统(SVN),用于哺乳动物细胞中大型多功能DNA电路的传递和精确对接。

DNA传递是基因治疗和合成生物学当前研究工作的最前沿。病毒载体传统上一直占据主导地位。但是,非病毒传递系统越来越受到关注。杆状病毒是节肢动物特异的病毒,可以很容易地进行工程改造和重新利用,以适应外源DNA的大序列传递并将其传递到哺乳动物细胞,组织或最终生物中。这些合成的病毒衍生纳米系统(SVN)是安全的,易于定制的,并且可以大规模生产。通过将成簇的规则间隔的回文重复(CRISPR)相关蛋白(CRISPR / Cas)模式应用于该系统,我们开发了能够以碱基对精度将复杂DNA插入基因组的SVN。
更新日期:2020-08-11
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