The generation and phenotypic assessment of mouse models for Rett Syndrome
The Jackson Laboratory is a renowned mouse genetics research institute and repository, dedicated to the development of robust mouse models and services for nonclinical drug testing. This proposal leverages their existing infrastructure and expertise to 1) genetically engineer MECP2 mutations into mice, 2) develop assays to detect RTT symptoms in the mouse models generated; 3) define the progression of Rett pathology in the mouse models.
This project will provide a great resource to Rett researchers by establishing a central facility to create comparable RTT mouse models, test potential therapeutic agents rigorously in a standardized, non-biased environment, and in the long run save time, money and funds.
We have established an advisory committee including expert scientists from the pioneering Bird, Greenberg, and Mandel groups plus several companies to help guide this project. John Sinnamon of the Mandel lab will serve as project manager.