期刊
PHARMACEUTICS
卷 12, 期 8, 页码 -出版社
MDPI
DOI: 10.3390/pharmaceutics12080759
关键词
baculovirus; CRISPR; gene editing; genome engineering; precision DNA docking
资金
- GE Healthcare
- BrisSynBio a BBSRC/EPSRC Research Centre for synthetic biology at the University of Bristol [BB/L01386X/1]
- Max Planck Bristol Centre for Minimal Biology
- European Research Council (ERC Advanced Grant DNA-DOCK) [834631]
- BBSRC [BB/L01386X/1] Funding Source: UKRI
- European Research Council (ERC) [834631] Funding Source: European Research Council (ERC)
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.
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