422094 Crispr-Based Self-Cleaving Mechanism for Controllable Gene Delivery in Human Cells

Wednesday, November 11, 2015
Exhibit Hall 1 (Salt Palace Convention Center)
Richard Moore, Bioengineering, University of Texas at Dallas, Richardson, TX and Leonidas Bleris, Electrical Engineeering, University of Texas at Dallas, Richardson, TX

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.

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See more of this Session: Poster Session: Bioengineering
See more of this Group/Topical: Food, Pharmaceutical & Bioengineering Division