Funded Research

Enabling Applied Genomic Technologies in Pediatric Diseases

Stewart, Sheila A. Ph.D.

PI Departments:
Cell Biology & Physiology
Collaborators:
Elaine Mardis,PhD and David Piwnica-Worms, MD, PhD
Award Mechanism:
Large Initiative
Project Period:
7/1/10-6/30/13
Lab/PI Website:
Project Summary:
The study of “model organisms” has led to an understanding of many basic cellular processes. One reason the study of these organisms has proven so fruitful is the ability to carry out genetic screens. While these studies have had an immense impact in our understanding of diverse cellular processes, one drawback is that many complex processes that impact human disease are not recapitulated in the model organisms. Fortunately, recent technical advances now allow us to carryout genetic screens (including the use of RNAi interference (RNAi)), ushering in a new area of scientific discovery. Despite the power of this technology, many have difficulty utilizing them due to lack of expertise or cost. While effective, RNAi remains expensive, and when a genome-wide approach is needed, it is cost-prohibitive. Here at Washington University, the CDI’s insightful investment has reduced the cost of human-specific RNAi, enabling the Washington University community access to this critical reagent. We propose to build an analogous resource directed against the mouse genome thus providing the Washington University community with a critical tool with which to study human disease. In addition, we will create a viral resource consisting of functional groups (i.e. kinases) whose disruption is known to impact a wide variety of human diseases. The creation of this critical resource will allow researchers the opportunity to bring this incredibly powerful technology to bear on pediatric diseases that will lead to a better understanding of disease and the identification of novel therapeutic targets.
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