A Generic, High-Throughput Screening Platform for Fragile X, Rett Syndrome, and Frontotemporal Degeneration Using Human Synthetic Embryos
Abstract
Fragile X, Rett syndrome, and frontotemporal degeneration are three devastating, rare neurological disorders that lack effective treatments, with authorized drugs that do exist mainly focusing on managing symptoms. In this proposal, we propose to develop a tool that will allow the efficient discovery of new therapeutics for these three diseases. Our approach is to recreate, in the laboratory, the first steps of the development of the central nervous system from embryonic stem cells. When these stem cells are carrying genetic mutation associated with one of these diseases, we can easily observe defects in the organization of our developing tissues. These differences between “healthy” and “disease” tissues are analyzed and exploited to find drugs that can reverse these defects. We are convinced that drugs that can cure synthetic human developing tissues in the lab will then translate efficiently in the clinic for curing patients.
Document Details
- Document Type
- DoD Grant Award
- Publication Date
- Mar 10, 2021
- Source ID
- W81XWH2010060
Entities
People
- Fred Etoc
Organizations
- Rumi Scientific (United States)
- United States Army