Science Webinar Series

Webinar | CRISPR/Cas9 in drug discovery: Applications in target discovery, validation, and hit screening

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Science  06 May 2016:
Vol. 352, Issue 6286, pp. 730
DOI: 10.1126/science.352.6286.730-b

Summary

The CRISPR/Cas9 system allows for unprecedented ease and control when editing the genome. Its potential impact on drug discovery is vast, including enabling gene and cell replacement therapies, identifying novel drug targets through functional genomic screens, and simplifying the production of disease models using permanent knockouts for validating therapy targets and testing drug efficacy. But in practical terms, how is CRISPR/Cas9 currently being applied, and where might the future challenges and pitfalls be? Furthermore, how do assays based on the new CRISPR/Cas9 technology compare with current screening methodologies, particularly those using small interfering RNA (siRNA)?