Yang, Wang-Yong

Small Molecules To Target r(CGG) Expansions to Treat Fragile X Syndrome

FRAXA Research Foundation 2012 Grants • 2013 Grants • Disney, Matthew • FRAXA Research Grants • Genetic Rescue • Research Grant Results • Treatment Targets • Yang, Wang-Yong January 24, 2014July 29, 2020amyloid precursor protein, FXTAS, mRNAs, NIH, RNAi

With a 2-year, $90,000 grant from FRAXA Research Foundation, Dr.’s Matthew Disney and Wang-Yong Yang worked to correct the underlying problem in Fragile X: the silencing of the Fragile X gene (FMR1) and the resulting lack of FMRP (Fragile X Mental Retardation Protein). Their approach was to use novel small molecules to target the abnormal CGG repeats before the FMR1 gene.