Harnessing the Potential of Genome Editing for Drug Discovery: Translational Frontiers of in vitro & in vivo Applications

 

Symposium - New York, United States

Until recently, the generation of precise alterations in mammalian genomes has been a challenging and cumbersome process. A rapid succession of technologies in the past several years has brought about a revolution in molecular biology: zinc-finger nucleases (ZFN), transcription activator-like effector nucleases (TALEN) and then clustered regularly interspaced short palindromic repeats (CRISPR). Each successive advancement has brought us closer to a routine, bench-level method to modify endogenous genetic elements. Now proof-of-concepts abound and encompass exciting frontiers such as rapid animal model development in addition to knockout and knock-in human and mouse cell lines. More recently, the framework of TALEN and CRISPR has been extended to a vast array of applications including epigenetic modulation, stimulation of gene expression, and targeted repression of gene transcription. What do these advances mean for drug discovery and translational research? This symposium will cover the latest techniques and methodologies in genome editing and the potential they hold for therapeutic development. In particular, the union of CRISPR and TALEN with stem and iPS cell technologies is an avenue that holds promise in improving the physiological relevance of disease models. However, the application of TALEN and CRIPSR to drug discovery is still in a nascent stage and innovation will be a core theme as we explore opportunities and current limitations.

 

Tue, Feb 24, 2015 - Tue, Feb 24, 2015 (08:30 - 16:30)

 

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