CRISPR-Based Self-Cleaving Mechanism for Controllable Gene Delivery in Human Cells

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CRISPR-Based Self-Cleaving Mechanism for Controllable Gene Delivery in Human Cells

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Title: CRISPR-Based Self-Cleaving Mechanism for Controllable Gene Delivery in Human Cells
Author(s):
Moore, Richard;
Spinhirne, Alec;
Lai, Michael J.;
Preisser, Samantha;
Li, Yi;
Kang, Taek;
Bleris, Leonidas
Item Type: article
Keywords: Show Keywords
Description: Supplementary materials are available at Nucleic Acids Research Online (see DOI).
Abstract: Controllable gene delivery via vector-based systems remains a formidable challenge in mammalian synthetic biology and a desirable asset in gene therapy applications. Here, we introduce a methodology to control the copies and residence time of a gene product delivered in host human cells but also selectively disrupt fragments of the delivery vehicle. A crucial element of the proposed system is the CRISPR protein Cas9. Upon delivery, Cas9 guided by a custom RNA sequence cleaves the delivery vector at strategically placed targets thereby inactivating a co-expressed gene of interest. Importantly, using experiments in human embryonic kidney cells, we show that specific parameters of the system can be adjusted to fine-tune the delivery properties. We envision future applications in complex synthetic biology architectures, gene therapy and trace-free delivery.;
Publisher: Oxford University Press
ISSN: 1362-4962
Persistent Link: http://dx.doi.org/10.1093/nar/gku1326
http://hdl.handle.net/10735.1/4396
Terms of Use: CC-BY-NC 4.0 (Attribution-Non-commercial use)
©2014 The Authors
Sponsors: US National Institutes of Health (GM098984, GM096271, CA17001801); US National Science Foundation (CBNET-1105524)

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CC-BY-NC 4.0 (Attribution-Non-commercial use) Except where otherwise noted, this item's license is described as CC-BY-NC 4.0 (Attribution-Non-commercial use)