Which is the right FMRP for Therapeutic Development of Fragile X Syndrome?
Samie Jaffrey, PhD
Principal Investigator
Wen-Biao Gan, PhD
Co-Investigator
Jiahui Wu, PhD
Postdoctoral Fellow
from Weill Medical College of Cornell University
New York, NY
2016-2017 Grant Funding: $90,000
Summary
Normally there are many forms of the Fragile X protein (FMRP) in brain cells. Dr. Jaffrey is working to identify the forms that are most important as treatment targets.
The Science
A major challenge in developing therapeutics for Fragile X syndrome is the fact that normally there are many forms of the Fragile X protein (FMRP). This one protein regulates various pathways in different cell compartments of both neuronal and non-neuronal cells. It is difficult to determine which FMRP-regulated pathway needs to be corrected to improve clinical outcomes in FXS.
To definitively answer the above questions, we propose to develop a highly robust method to selectively restore protein expression in a compartment-specific and cell-specific
manner of live animals and determine if this reverses synaptic and spine defects. Our long-term goal is to indentify druggable targets for FXS therapeutic development.
