CRISPR-Based Self-Cleaving Mechanism for Controllable Gene Delivery in Human Cells
dc.contributor.ISNI | 0000 0001 2535 9739 (Bleris, L) | en_US |
dc.contributor.LCNA | 2012076942 (Bleris, L) | en_US |
dc.contributor.author | Moore, Richard | en_US |
dc.contributor.author | Spinhirne, Alec | en_US |
dc.contributor.author | Lai, Michael J. | en_US |
dc.contributor.author | Preisser, Samantha | en_US |
dc.contributor.author | Li, Yi | en_US |
dc.contributor.author | Kang, Taek | en_US |
dc.contributor.author | Bleris, Leonidas | en_US |
dc.date.accessioned | 2015-03-30T15:25:28Z | |
dc.date.available | 2015-03-30T15:25:28Z | |
dc.date.created | 2014-12-18 | en_US |
dc.date.issued | 2014-12-18 | en_US |
dc.description | Supplementary materials are available at Nucleic Acids Research Online (see DOI). | en_US |
dc.description.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.; | en_US |
dc.description.sponsorship | US National Institutes of Health (GM098984, GM096271, CA17001801); US National Science Foundation (CBNET-1105524) | en_US |
dc.identifier.bibliographicCitation | Moore, Richard, Alec Spinhime, Michael J. Lai, Samantha Preisser, et al. 2014. "CRISPR-based self-cleaving mechanism for controllable gene delivery in human cells." Nucleic Acids Research 43(2): doi:10.1093/nar/gku1326. | |
dc.identifier.issn | 1362-4962 | en_US |
dc.identifier.uri | http://hdl.handle.net/10735.1/4396 | |
dc.publisher | Oxford University Press | en_US |
dc.relation.uri | http://dx.doi.org/10.1093/nar/gku1326 | en_US |
dc.rights | CC BY-NC 4.0 (Attribution-Non-commercial) | en_US |
dc.rights | ©2014 The Authors | en_US |
dc.rights.uri | http://creativecommons.org/licenses/by-nc/4.0/ | en_US |
dc.source.journal | Nucleic Acids Research | en_US |
dc.subject | mKate2 protein | en_US |
dc.subject | Cas9 protein | en_US |
dc.subject | Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) | en_US |
dc.subject | Guide RNA (gRNA) | en_US |
dc.subject | Gene delivery | en_US |
dc.title | CRISPR-Based Self-Cleaving Mechanism for Controllable Gene Delivery in Human Cells | en_US |
dc.type.genre | article | en_US |
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