RNAi and CRISPR/Cas9-Based In Vivo Models for Drug Discovery

Apr 23 2015

Genetically engineered mouse models (GEMMs) are a powerful platform for enabling the study of disease initiation and maintenance, the microenvironment and the responsiveness of disease to known or novel therapeutics. However, the long lead times and high costs required to develop, intercross and maintain models with various gene combinations have limited their practical utility in the drug discovery process. RNA interference (RNAi) is a rapid, cost-effective alternative to gene deletion that can be exploited experimentally to reversibly silence nearly any gene target not only in vitro, but also in live mice. Mirimus has developed a fast, scalable pipeline for the production of short hairpin RNA (shRNA) transgenic mice with potent reversible gene-silencing potential.

With the advent of new genome editing techniques such as CRISPR/Cas9, it has become possible to introduce additional sensitizing lesions to induce disease pathogenesis. In synergy with RNAi technology, complex multi-allelic ESC-based GEMMs can be generated without extensive intercrossing, resulting in the ability to model disease pathogenesis and mimic drug therapy in mice and enabling unprecedented capabilities for performing preclinical studies in vivo.

This webinar demonstrates how Mirimus’ RNAi technology, in combination with CRISPR/Cas9 genome editing, facilitates recapitulation of knockout mice phenotypes and further exploration of potential therapeutic approaches within the same model. This robust system provides a cost-effective, scalable platform for the production of mice with enormous predictive power that will shape development of better-tolerated therapies.


  • Prem K. Premsrirut, PhD, Co-founder, President and CEO, Mirimus, Inc.