MU Rodent Testing Center for Somatic Cell Genome Editing

PROJECT SUMMARY: OVERALL
Advances in genome editing have paved the way for the possibility of new therapeutic strategies for treating
human disease. There is a current need for animal resources and services that can assist with studies that will
accelerate the translation of genome editing technology into clinical applications. In particular, the availability of
a center that can provide testing not only in mice but in rats, which are often a preferred model species for
validation of therapeutics, is greatly needed. The MU Rodent Testing Center for Somatic Cell Genome Editing
brings together a team with longstanding and established expertise in the fields of animal modeling and
comparative medicine who are equally proficient at working with both mice and rats. Additionally, strengths of
the assembled collaborative team include 1) access to large numbers of rodent disease models and reporter
strains and the demonstrated ability to acquire/cryorecover/make any needed mouse/rat strain, 2) expertise in
the maintenance and distribution of rodents under the highest quality standards to ensure downstream research
rigor and reproducibility, 3) access to established and state-of-the art infrastructure and phenotyping expertise
in rodents, 4) experience operating facilities that are service-oriented and familiar with managing fee-for-service
operations, and 5) the demonstrated ability to work successfully together and as part of collaborative
interdisciplinary research teams. The specific goals of the MU Rodent Testing Center for Somatic Cell Genome
Editing are 1) breed and maintain groups of wild-type, reporter and disease model mice and rats for in vivo
testing, 2) establish assays and protocols for evaluating on and off-target genome editing, biodistribution,
bioactivity, and safety using a variety of delivery methods, including in utero delivery, and 3) provide the
resources and services needed by biomedical researchers to evaluate new reagents/tools related to somatic cell
genome editing/gene therapy in relevant preclinical rodent models. Our collaborative group can work with
investigators to provide comprehensive evaluation of somatic cell genome editing tools and delivery vehicles in
whole animals and embryos as well as in all tissues and cell types. The MU Rodent Testing Center for Somatic
Cell Genome Editing is ideally suited to provide the rodent-based expertise needed to facilitate the ability to
evaluate new genome editing tools and technologies for future application to human disease treatments.

Grant Number: 5U42OD035739-02
NIH Institute/Center: NIH
Principal Investigator: Elizabeth Bryda